Paul Harmatz, MD

Title(s)Professor, Pediatrics
SchoolSchool of Medicine
Address744 52nd Street, #001
Oakland CA
Phone--
ORCID ORCID Icon0000-0001-5603-2085 Additional info
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    Collapse Biography 
    Collapse Education and Training
    Stanford University, Palo Alto, CAAB06/1973Human Biology
    Dartmouth Medical School, Hanover, NHMD06/1976Medicine
    Harbor UCLA, Torrance, CA06/1979Pediatric Residency
    Massachusetts General Hospital, Boston, MA06/1982Fellowship Pediatric Gastroenterology and Nutrition

    Collapse Overview 
    Collapse Overview
    Dr. Paul Harmatz is a gastroenterologist who specializes in mucopolysaccharidoses (MPS) and other lysosomal storage diseases (genetic disorders in which a lack of certain enzymes results in progressive damage to cells and organ systems). He leads a team of specialists who diagnose and care for patients with these rare diseases, offering therapies such as weekly enzyme infusion.

    Harmatz's research focuses in particular on promising new treatments for MPS disease. He has led clinical trials to evaluate treatments for a variety of MPS and other lysosomal storage diseases, resulting in approval from the Food and Drug Administration for five specific treatments. He also led the first trial on a drug to treat mucopolysaccharidosis type VI (also called Maroteaux-Lamy syndrome or MPS VI). In addition, he led a trial in which genome editing was done inside a human body for the first time in an effort to change the DNA of a patient with mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II).

    Harmatz earned his medical degree from Dartmouth Geisel School of Medicine. He completed a residency in pediatrics at Harbor – UCLA Medical Center, followed by a fellowship in pediatric gastroenterology and nutrition at Massachusetts General Hospital. Prior to joining UCSF, he served on the faculty of Harvard Medical School for 12 years.

    Harmatz is a member of the Society for the Study of Inborn Errors of Metabolism, Society for Inherited Metabolic Disorders and American Society of Gene & Cell Therapy. In 2017, UCSF awarded him a medical science career achievement award.

    Collapse Research 
    Collapse Research Activities and Funding
    TRANSPORT OF IGA IMMUNE COMPLEXES INTO BREAST MILK
    NIH K08AM001141Apr 1, 1983 - Mar 31, 1986
    Role: Principal Investigator

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    Collapse Clinical Trials

    Collapse Bibliographic 
    Collapse Publications
    Publications listed below are automatically derived from MEDLINE/PubMed and other sources, which might result in incorrect or missing publications. Researchers can login to make corrections and additions, or contact us for help. to make corrections and additions.
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    Altmetrics Details PMC Citations indicate the number of times the publication was cited by articles in PubMed Central, and the Altmetric score represents citations in news articles and social media. (Note that publications are often cited in additional ways that are not shown here.) Fields are based on how the National Library of Medicine (NLM) classifies the publication's journal and might not represent the specific topic of the publication. Translation tags are based on the publication type and the MeSH terms NLM assigns to the publication. Some publications (especially newer ones and publications not in PubMed) might not yet be assigned Field or Translation tags.) Click a Field or Translation tag to filter the publications.
    1. α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial. Mol Ther. 2024 Mar 06; 32(3):609-618. Harmatz P, Giugliani R, Martins AM, Hamazaki T, Kubo T, Kira R, Minami K, Ikeda T, Moriuchi H, Kawashima S, Takasao N, So S, Sonoda H, Hirato T, Tanizawa K, Schmidt M, Sato Y. PMID: 38204164; PMCID: PMC10928130.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCTClinical Trials
    2. Biomarkers of Glycosaminoglycans (GAG) accumulation in patients with mucopolysaccharidosis type VI-LeukoGAG, Corneal Opacification (COM) and Carotid Intima Media Thickening (CIMT). Mol Genet Metab Rep. 2024 Mar; 38:101041. Sohn YB, Wang R, Ashworth J, Broqua P, Tallandier M, Abitbol JL, Jozwiak E, Pollard L, Wood TC, Aslam T, Harmatz PR. PMID: 38234862; PMCID: PMC10792263.
      View in: PubMed   Mentions:
    3. Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Child Adolesc Health. 2024 Jan; 8(1):40-50. Savarirayan R, Wilcox WR, Harmatz P, Phillips J, Polgreen LE, Tofts L, Ozono K, Arundel P, Irving M, Bacino CA, Basel D, Bober MB, Charrow J, Mochizuki H, Kotani Y, Saal HM, Army C, Jeha G, Qi Y, Han L, Fisheleva E, Huntsman-Labed A, Day J. PMID: 37984383.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCTClinical Trials
    4. Neurodevelopmental status and adaptive behavior of pediatric patients with mucopolysaccharidosis II: a longitudinal observational study. Orphanet J Rare Dis. 2023 Nov 16; 18(1):357. Muenzer J, Burton BK, Amartino HM, Harmatz PR, Gutiérrez-Solana LG, Ruiz-Garcia M, Wu Y, Merberg D, Alexanderian D, Jones SA. PMID: 37974184; PMCID: PMC10652632.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    5. Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects. Prenat Diagn. 2023 12; 43(13):1638-1649. Herzeg A, Borges B, Lianoglou BR, Gonzalez-Velez J, Canepa E, Munar D, Young SP, Bali D, Gelb MH, Chakraborty P, Kishnani PS, Harmatz P, Cohen JL, MacKenzie TC. PMID: 37955580.
      View in: PubMed   Mentions:    Fields:    Translation:HumansCells
    6. Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey. Mol Genet Metab Rep. 2023 Dec; 37:101005. Lau H, Harmatz P, Botha J, Audi J, Link B. PMID: 38053935; PMCID: PMC10694755.
      View in: PubMed   Mentions:
    7. Corrigendum to: Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study. Mol Genet Metab. 2023 Nov; 140(3):107645. Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, HGT-HIT-094 Study Group. PMID: 37541906.
      View in: PubMed   Mentions:    Fields:    
    8. Corrigendum to "Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II". Mol Genet Metab. 2023 Nov; 140(3):107646. Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, SHP609-302 study group. PMID: 37517985.
      View in: PubMed   Mentions:    Fields:    
    9. Growth patterns in patients with mucopolysaccharidosis VII. Mol Genet Metab Rep. 2023 Sep; 36:100987. Montaño AM, Rózdzynska-Swiatkowska A, Jurecka A, Ramirez AN, Zhang L, Marsden D, Wang RY, Harmatz P. PMID: 37415957; PMCID: PMC10320588.
      View in: PubMed   Mentions:
    10. Persistent bone and joint disease despite current treatments for mucopolysaccharidosis types I, II, and VI: Data from a 10-year prospective study. J Inherit Metab Dis. 2023 07; 46(4):695-704. Miller BS, Fung EB, White KK, Lund TC, Harmatz P, Orchard PJ, Whitley CB, Polgreen LE. PMID: 36840680; PMCID: PMC10824391.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    11. A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B. J Clin Invest. 2023 01 17; 133(2). Muschol N, Koehn A, von Cossel K, Okur I, Ezgu F, Harmatz P, de Castro Lopez MJ, Couce ML, Lin SP, Batzios S, Cleary M, Solano M, Nestrasil I, Kaufman B, Shaywitz AJ, Maricich SM, Kuca B, Kovalchin J, Zanelli E. PMID: 36413418; PMCID: PMC9843052.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCTClinical Trials
    12. In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease. N Engl J Med. 2022 12 08; 387(23):2150-2158. Cohen JL, Chakraborty P, Fung-Kee-Fung K, Schwab ME, Bali D, Young SP, Gelb MH, Khaledi H, DiBattista A, Smallshaw S, Moretti F, Wong D, Lacroix C, El Demellawy D, Strickland KC, Lougheed J, Moon-Grady A, Lianoglou BR, Harmatz P, Kishnani PS, MacKenzie TC. PMID: 36351280; PMCID: PMC10794051.
      View in: PubMed   Mentions: 16     Fields:    Translation:Humans
    13. Current and new therapies for mucopolysaccharidoses. Pediatr Neonatol. 2023 02; 64 Suppl 1:S10-S17. Penon-Portmann M, Blair DR, Harmatz P. PMID: 36464587.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    14. First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B. Mol Ther. 2022 12 07; 30(12):3587-3600. Harmatz P, Prada CE, Burton BK, Lau H, Kessler CM, Cao L, Falaleeva M, Villegas AG, Zeitler J, Meyer K, Miller W, Wong Po Foo C, Vaidya S, Swenson W, Shiue LH, Rouy D, Muenzer J. PMID: 36299240; PMCID: PMC9734078.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
    15. Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study. Genet Med. 2022 Dec; 24(12):2444-2452. Savarirayan R, Irving M, Harmatz P, Delgado B, Wilcox WR, Philips J, Owen N, Bacino CA, Tofts L, Charrow J, Polgreen LE, Hoover-Fong J, Arundel P, Ginebreda I, Saal HM, Basel D, Font RU, Ozono K, Bober MB, Cormier-Daire V, Le Quan Sang KH, Baujat G, Alanay Y, Rutsch F, Hoernschemeyer D, Mohnike K, Mochizuki H, Tajima A, Kotani Y, Weaver DD, White KK, Army C, Larrimore K, Gregg K, Jeha G, Milligan C, Fisheleva E, Huntsman-Labed A, Day J. PMID: 36107167.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    16. Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study. Mol Genet Metab. 2022 Sep-Oct; 137(1-2):127-139. Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, HGT-HIT-094 Study Group. PMID: 36027721; PMCID: PMC10826424.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansCTClinical Trials
    17. Long-term open-label extension study of the safety and efficacy of intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II. Mol Genet Metab. 2022 Sep-Oct; 137(1-2):92-103. Muenzer J, Burton BK, Harmatz P, Gutiérrez-Solana LG, Ruiz-Garcia M, Jones SA, Guffon N, Inbar-Feigenberg M, Bratkovic D, Hale M, Wu Y, Yee KS, Whiteman DAH, Alexanderian D, SHP609-302 study group. PMID: 35961250; PMCID: PMC10826456.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansCTClinical Trials
    18. Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study. Mol Genet Metab. 2022 08; 136(4):249-259. Harmatz P, Muenzer J, Ezgü F, Dalén P, Huledal G, Lindqvist D, Gelius SS, Wikén M, Önnestam K, Bröijersén A. PMID: 35835061.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    19. Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB. J Pediatr. 2022 10; 249:50-58.e2. Okur I, Ezgu F, Giugliani R, Muschol N, Koehn A, Amartino H, Harmatz P, de Castro Lopez MJ, Couce ML, Lin SP, Batzios S, Cleary M, Solano M, Peters H, Lee J, Nestrasil I, Shaywitz AJ, Maricich SM, Kuca B, Kovalchin J, Zanelli E. PMID: 35709957.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    20. RNA analysis of the GALNS transcript reveals novel pathogenic mechanisms associated with Morquio syndrome A. Mol Genet Metab Rep. 2022 Jun; 31:100875. Sohn YB, Rogers C, Stallworth J, Cooley Coleman JA, Buch L, Jozwiak E, Johnson JA, Wood T, Harmatz P, Pollard L, Louie RJ. PMID: 35782621; PMCID: PMC9248232.
      View in: PubMed   Mentions:
    21. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. Ther Adv Musculoskelet Dis. 2022; 14:1759720X221084848. Savarirayan R, De Bergua JM, Arundel P, McDevitt H, Cormier-Daire V, Saraff V, Skae M, Delgado B, Leiva-Gea A, Santos-Simarro F, Salles JP, Nicolino M, Rossi M, Kannu P, Bober MB, Phillips J, Saal H, Harmatz P, Burren C, Gotway G, Cho T, Muslimova E, Weng R, Rogoff D, Hoover-Fong J, Irving M. PMID: 35342457; PMCID: PMC8941703.
      View in: PubMed   Mentions: 8  
    22. Timing is everything: Clinical courses of Hunter syndrome associated with age at initiation of therapy in a sibling pair. Mol Genet Metab Rep. 2022 Mar; 30:100845. Grant N, Sohn YB, Ellinwood NM, Okenfuss E, Mendelsohn BA, Lynch LE, Braunlin EA, Harmatz PR, Eisengart JB. PMID: 35242576; PMCID: PMC8856919.
      View in: PubMed   Mentions: 5  
    23. Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients. Orphanet J Rare Dis. 2022 01 29; 17(1):25. Schwab ME, Brown JEH, Lianoglou B, Jin C, Conroy PC, Gallagher RC, Harmatz P, MacKenzie TC. PMID: 35093147; PMCID: PMC8800365.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    24. Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I. Mol Genet Metab. 2022 02; 135(2):122-132. Kovac V, Shapiro EG, Rudser KD, Mueller BA, Eisengart JB, Delaney KA, Ahmed A, King KE, Yund BD, Cowan MJ, Raiman J, Mamak EG, Harmatz PR, Shankar SP, Ali N, Cagle SR, Wozniak JR, Lim KO, Orchard PJ, Whitley CB, Nestrasil I. PMID: 35012890; PMCID: PMC8898074.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    25. Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients. Orphanet J Rare Dis. 2021 12 02; 16(1):500. Leiro B, Phillips D, Duiker M, Harmatz P, Charles S. PMID: 34857033; PMCID: PMC8638175.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    26. Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS). Orphanet J Rare Dis. 2021 10 30; 16(1):456. Muenzer J, Botha J, Harmatz P, Giugliani R, Kampmann C, Burton BK. PMID: 34717704; PMCID: PMC8557006.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    27. Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment. J Inherit Metab Dis. 2021 11; 44(6):1463-1480. Mengel E, Patterson MC, Da Riol RM, Del Toro M, Deodato F, Gautschi M, Grunewald S, Grønborg S, Harmatz P, Héron B, Maier EM, Roubertie A, Santra S, Tylki-Szymanska A, Day S, Andreasen AK, Geist MA, Havnsøe Torp Petersen N, Ingemann L, Hansen T, Blaettler T, Kirkegaard T, Í Dali C. PMID: 34418116; PMCID: PMC9293014.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCTClinical Trials
    28. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genet Med. 2021 12; 23(12):2443-2447. Savarirayan R, Tofts L, Irving M, Wilcox WR, Bacino CA, Hoover-Fong J, Font RU, Harmatz P, Rutsch F, Bober MB, Polgreen LE, Ginebreda I, Mohnike K, Charrow J, Hoernschemeyer D, Ozono K, Alanay Y, Arundel P, Kotani Y, Yasui N, White KK, Saal HM, Leiva-Gea A, Luna-González F, Mochizuki H, Basel D, Porco DM, Jayaram K, Fisheleva E, Huntsman-Labed A, Day JRS. PMID: 34341520; PMCID: PMC8327889.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCTClinical Trials
    29. Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy. Mol Genet Metab. 2021 05; 133(1):100-108. Garcia P, Phillips D, Johnson J, Martin K, Randolph LM, Rosenfeld H, Harmatz P. PMID: 33775523.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    30. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2020 09 05; 396(10252):684-692. Savarirayan R, Tofts L, Irving M, Wilcox W, Bacino CA, Hoover-Fong J, Ullot Font R, Harmatz P, Rutsch F, Bober MB, Polgreen LE, Ginebreda I, Mohnike K, Charrow J, Hoernschemeyer D, Ozono K, Alanay Y, Arundel P, Kagami S, Yasui N, White KK, Saal HM, Leiva-Gea A, Luna-González F, Mochizuki H, Basel D, Porco DM, Jayaram K, Fisheleva E, Huntsman-Labed A, Day J. PMID: 32891212.
      View in: PubMed   Mentions: 44     Fields:    Translation:HumansCTClinical Trials
    31. Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints. Mol Genet Metab. 2020 Sep - Oct; 131(1-2):181-196. van der Lee JH, Morton J, Adams HR, Clarke L, Eisengart JB, Escolar ML, Giugliani R, Harmatz P, Hogan M, Kearney S, Muenzer J, Muschol N, Rust S, Saville BR, Semrud-Clikeman M, Wang R, Shapiro E. PMID: 32917509.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    32. Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227]. Mol Genet Metab. 2020 Sep - Oct; 131(1-2):285. Wang RY, da Silva Franco JF, López-Valdez J, Martins E, Sutton VR, Whitley CB, Zhang L, Cimms T, Marsden D, Jurecka A, Harmatz P. PMID: 32843286.
      View in: PubMed   Mentions:    Fields:    
    33. ASAH1 pathogenic variants associated with acid ceramidase deficiency: Farber disease and spinal muscular atrophy with progressive myoclonic epilepsy. Hum Mutat. 2020 09; 41(9):1469-1487. Elsea SH, Solyom A, Martin K, Harmatz P, Mitchell J, Lampe C, Grant C, Selim L, Mungan NO, Guelbert N, Magnusson B, Sundberg E, Puri R, Kapoor S, Arslan N, DiRocco M, Zaki M, Ozen S, Mahmoud IG, Ehlert K, Hahn A, Gokcay G, Torcoletti M, Ferreira CR. PMID: 32449975.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansAnimals
    34. Assessing the impact of the five senses on quality of life in mucopolysaccharidoses. Orphanet J Rare Dis. 2020 04 19; 15(1):97. Giugliani R, Harmatz P, Lin SP, Scarpa M. PMID: 32306998; PMCID: PMC7168888.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    35. The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. Mol Genet Metab. 2020 03; 129(3):219-227. Wang RY, da Silva Franco JF, López-Valdez J, Martins E, Sutton VR, Whitley CB, Zhang L, Cimms T, Marsden D, Jurecka A, Harmatz P. PMID: 32063397.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansCTClinical Trials
    36. Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study. Mol Genet Metab. 2020 02; 129(2):80-90. Chen AH, Harmatz P, Nestrasil I, Eisengart JB, King KE, Rudser K, Kaizer AM, Svatkova A, Wakumoto A, Le SQ, Madden J, Young S, Zhang H, Polgreen LE, Dickson PI. PMID: 31839529; PMCID: PMC7813548.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    37. Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program. Mol Genet Metab. 2019 08; 127(4):355-360. Lampe C, Harmatz PR, Parini R, Sharma R, Teles EL, Johnson J, Sivam D, Sisic Z. PMID: 31324526.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    38. Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study. J Clin Endocrinol Metab. 2019 07 01; 104(7):2735-2747. Hofmann CE, Harmatz P, Vockley J, Högler W, Nakayama H, Bishop N, Martos-Moreno GÁ, Moseley S, Fujita KP, Liese J, Rockman-Greenberg C, ENB-010-10 Study Group. PMID: 30811537; PMCID: PMC6530655.
      View in: PubMed   Mentions: 21     Fields:    Translation:HumansCTClinical Trials
    39. Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population. JIMD Rep. 2019 Sep; 49(1):53-62. Haller C, Song W, Cimms T, Chen CY, Whitley CB, Wang RY, Bauer M, Harmatz P. PMID: 31497482; PMCID: PMC6718107.
      View in: PubMed   Mentions: 6  
    40. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. N Engl J Med. 2019 07 04; 381(1):25-35. Savarirayan R, Irving M, Bacino CA, Bostwick B, Charrow J, Cormier-Daire V, Le Quan Sang KH, Dickson P, Harmatz P, Phillips J, Owen N, Cherukuri A, Jayaram K, Jeha GS, Larimore K, Chan ML, Huntsman Labed A, Day J, Hoover-Fong J. PMID: 31269546.
      View in: PubMed   Mentions: 63     Fields:    Translation:HumansCTClinical Trials
    41. Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. Orphanet J Rare Dis. 2019 06 13; 14(1):137. Akyol MU, Alden TD, Amartino H, Ashworth J, Belani K, Berger KI, Borgo A, Braunlin E, Eto Y, Gold JI, Jester A, Jones SA, Karsli C, Mackenzie W, Marinho DR, McFadyen A, McGill J, Mitchell JJ, Muenzer J, Okuyama T, Orchard PJ, Stevens B, Thomas S, Walker R, Wynn R, Giugliani R, Harmatz P, Hendriksz C, Scarpa M, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs. PMID: 31196221; PMCID: PMC6567385.
      View in: PubMed   Mentions: 35     Fields:    Translation:Humans
    42. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. Orphanet J Rare Dis. 2019 05 29; 14(1):118. Akyol MU, Alden TD, Amartino H, Ashworth J, Belani K, Berger KI, Borgo A, Braunlin E, Eto Y, Gold JI, Jester A, Jones SA, Karsli C, Mackenzie W, Marinho DR, McFadyen A, McGill J, Mitchell JJ, Muenzer J, Okuyama T, Orchard PJ, Stevens B, Thomas S, Walker R, Wynn R, Giugliani R, Harmatz P, Hendriksz C, Scarpa M, MPS Consensus Programme Steering Committee, MPS Consensus Programme Co-Chairs. PMID: 31142378; PMCID: PMC6541999.
      View in: PubMed   Mentions: 15     Fields:    Translation:Humans
    43. Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type I. Neurology. 2019 05 14; 92(20):e2321-e2328. King KE, Rudser KD, Nestrasil I, Kovac V, Delaney KA, Wozniak JR, Mueller BA, Lim KO, Eisengart JB, Mamak EG, Raiman J, Ali N, Cagle S, Harmatz P, Whitley CB, Shapiro EG. PMID: 30979856; PMCID: PMC6598818.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    44. Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program. J Inherit Metab Dis. 2019 05; 42(3):519-526. Harmatz PR, Lampe C, Parini R, Sharma R, Teles EL, Johnson J, Sivam D, Sisic Z. PMID: 30834539.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    45. Spinal cord issues in adult patients with MPS: transition of care survey. Childs Nerv Syst. 2018 09; 34(9):1759-1765. Ghotme KA, Alvarado-Gomez F, Lampe C, White KK, Solano-Villareal M, Giugliani R, Harmatz PR. PMID: 29804213.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    46. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. J Inherit Metab Dis. 2018 11; 41(6):1225-1233. Lund AM, Borgwardt L, Cattaneo F, Ardigò D, Geraci S, Gil-Campos M, De Meirleir L, Laroche C, Dolhem P, Cole D, Tylki-Szymanska A, Lopez-Rodriguez M, Guillén-Navarro E, Dali CI, Héron B, Fogh J, Muschol N, Phillips D, Van den Hout JMH, Jones SA, Amraoui Y, Harmatz P, Guffon N. PMID: 29725868; PMCID: PMC6326957.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCTClinical Trials
    47. Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis. Mol Genet Metab. 2018 06; 124(2):152-160. Harmatz P, Cattaneo F, Ardigò D, Geraci S, Hennermann JB, Guffon N, Lund A, Hendriksz CJ, Borgwardt L. PMID: 29716835.
      View in: PubMed   Mentions: 12     Fields:    Translation:Humans
    48. Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B. J Pediatr. 2018 06; 197:198-206.e2. Whitley CB, Cleary M, Eugen Mengel K, Harmatz P, Shapiro E, Nestrasil I, Haslett P, Whiteman D, Alexanderian D. PMID: 29661560.
      View in: PubMed   Mentions: 11     Fields:    Translation:HumansCTClinical Trials
    49. Risks of long-term port use in enzyme replacement therapy for lysosomal storage disorders. Mol Genet Metab Rep. 2018 Jun; 15:71-73. Hendriksz CJ, Harmatz P, Giugliani R, Roberts J, Arul GS. PMID: 29845015; PMCID: PMC5966583.
      View in: PubMed   Mentions: 3  
    50. A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease. Mol Genet Metab. 2018 04; 123(4):488-494. Harmatz P, Whitley CB, Wang RY, Bauer M, Song W, Haller C, Kakkis E. PMID: 29478819.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCTClinical Trials
    51. Intrafamilial variability in the clinical manifestations of mucopolysaccharidosis type II: Data from the Hunter Outcome Survey (HOS). Am J Med Genet A. 2018 02; 176(2):301-310. Ficicioglu C, Giugliani R, Harmatz P, Mendelsohn NJ, Jego V, Parini R. PMID: 29210515; PMCID: PMC5814921.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    52. Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study. Mol Genet Metab. 2018 02; 123(2):127-134. Hendriksz CJ, Parini R, AlSayed MD, Raiman J, Giugliani R, Mitchell JJ, Burton BK, Guelbert N, Stewart FJ, Hughes DA, Matousek R, Hawley SM, Decker C, Harmatz PR. PMID: 29248359.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCTClinical Trials
    53. Cardiopulmonary Exercise Testing Reflects Improved Exercise Capacity in Response to Treatment in Morquio A Patients: Results of a 52-Week Pilot Study of Two Different Doses of Elosulfase Alfa. JIMD Rep. 2018; 42:9-17. Berger KI, Burton BK, Lewis GD, Tarnopolsky M, Harmatz PR, Mitchell JJ, Muschol N, Jones SA, Sutton VR, Pastores GM, Lau H, Sparkes R, Shaywitz AJ. PMID: 29159458; PMCID: PMC6226396.
      View in: PubMed   Mentions: 3  
    54. Surgical management of neurological manifestations of mucopolysaccharidosis disorders. Mol Genet Metab. 2017 12; 122S:41-48. Alden TD, Amartino H, Dalla Corte A, Lampe C, Harmatz PR, Vedolin L. PMID: 29153846.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCells
    55. Monitoring guidance for patients with hypophosphatasia treated with asfotase alfa. Mol Genet Metab. 2017 09; 122(1-2):4-17. Kishnani PS, Rush ET, Arundel P, Bishop N, Dahir K, Fraser W, Harmatz P, Linglart A, Munns CF, Nunes ME, Saal HM, Seefried L, Ozono K. PMID: 28888853.
      View in: PubMed   Mentions: 46     Fields:    Translation:Humans
    56. Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure. Mol Genet Metab. 2017 06; 121(2):70-79. van der Lee JH, Morton J, Adams HR, Clarke L, Ebbink BJ, Escolar ML, Giugliani R, Harmatz P, Hogan M, Jones S, Kearney S, Muenzer J, Rust S, Semrud-Clikeman M, Wijburg FA, Yu ZF, Janzen D, Shapiro E. PMID: 28501294.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    57. Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry. Orphanet J Rare Dis. 2017 05 02; 12(1):82. Muenzer J, Jones SA, Tylki-Szymanska A, Harmatz P, Mendelsohn NJ, Guffon N, Giugliani R, Burton BK, Scarpa M, Beck M, Jangelind Y, Hernberg-Stahl E, Larsen MP, Pulles T, Whiteman DAH. PMID: 28464912; PMCID: PMC5414331.
      View in: PubMed   Mentions: 28     Fields:    Translation:Humans
    58. The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Mol Genet Metab. 2017 09; 122(1-2):107-112. Harmatz P, Hendriksz CJ, Lampe C, McGill JJ, Parini R, Leão-Teles E, Valayannopoulos V, Cole TJ, Matousek R, Graham S, Guffon N, Quartel A, MPS VI Study Group. PMID: 28457718.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCTClinical Trials
    59. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients. Mol Genet Metab Rep. 2017 Sep; 12:2-7. Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. PMID: 28243577; PMCID: PMC5320046.
      View in: PubMed   Mentions: 10  
    60. Mucopolysaccharidosis VI: pathophysiology, diagnosis and treatment. Front Biosci (Landmark Ed). 2017 01 01; 22(3):385-406. Harmatz P, Shediac R. PMID: 27814620.
      View in: PubMed   Mentions: 38     Fields:    Translation:Humans
    61. Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study. Clin Ther. 2017 Jan; 39(1):118-129.e3. Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B. PMID: 27955919.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCTClinical Trials
    62. Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study. Am J Med Genet A. 2017 Feb; 173(2):375-383. Harmatz PR, Mengel E, Geberhiwot T, Muschol N, Hendriksz CJ, Burton BK, Jameson E, Berger KI, Jester A, Treadwell M, Sisic Z, Decker C. PMID: 27774754; PMCID: PMC5298029.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    63. Pregnancy in patients with mucopolysaccharidosis: a case series. Mol Genet Metab Rep. 2016 Sep; 8:111-5. Stewart FJ, Bentley A, Burton BK, Guffon N, Hale SL, Harmatz PR, Kircher SG, Kochhar PK, Mitchell JJ, Plöckinger U, Graham S, Sande S, Sisic Z, Johnston TA. PMID: 27622143; PMCID: PMC5007877.
      View in: PubMed   Mentions: 8  
    64. Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome. J Inherit Metab Dis. 2016 11; 39(6):839-847. Hendriksz CJ, Berger KI, Parini R, AlSayed MD, Raiman J, Giugliani R, Mitchell JJ, Burton BK, Guelbert N, Stewart F, Hughes DA, Matousek R, Jurecki E, Decker C, Harmatz PR. PMID: 27553181; PMCID: PMC5065598.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCTClinical Trials
    65. Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome. Mol Genet Metab. 2016 09; 119(1-2):131-43. Hendriksz CJ, Parini R, AlSayed MD, Raiman J, Giugliani R, Solano Villarreal ML, Mitchell JJ, Burton BK, Guelbert N, Stewart F, Hughes DA, Berger KI, Slasor P, Matousek R, Jurecki E, Shaywitz AJ, Harmatz PR. PMID: 27380995.
      View in: PubMed   Mentions: 36     Fields:    Translation:HumansCTClinical Trials
    66. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Mol Genet Metab. 2016 08; 118(4):310-8. Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR, CSP Study Group. PMID: 27339555.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCTClinical Trials
    67. The natural history of growth in patients with Hunter syndrome: Data from the Hunter Outcome Survey (HOS). Mol Genet Metab. 2016 Apr; 117(4):438-46. Parini R, Jones SA, Harmatz PR, Giugliani R, Mendelsohn NJ. PMID: 26846156.
      View in: PubMed   Mentions: 28     Fields:    Translation:Humans
    68. Implementing evidence-driven individualized treatment plans within Morquio A Syndrome. Mol Genet Metab. 2016 Feb; 117(2):217. Clarke LA, Harmatz P, Fong EW. PMID: 26877092.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    69. Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y. Pediatr Res. 2015 Dec; 78(6):717-22. Jones SA, Bialer M, Parini R, Martin K, Wang H, Yang K, Shaywitz AJ, Harmatz P. PMID: 26331768; PMCID: PMC4700045.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCTClinical Trials
    70. Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I. Data Brief. 2015 Dec; 5:71-6. Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE, Mucopolysaccharidosis I Intrathecal Research Collaborative. PMID: 26484358; PMCID: PMC4573094.
      View in: PubMed   Mentions: 4  
    71. Enzyme Replacement Therapies and Immunogenicity in Lysosomal Storage Diseases: Is There a Pattern? Clin Ther. 2015 Sep; 37(9):2130-4. Harmatz P. PMID: 26243075.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    72. Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I. Mol Genet Metab. 2015 Sep-Oct; 116(1-2):69-74. Dickson PI, Kaitila I, Harmatz P, Mlikotic A, Chen AH, Victoroff A, Passage MB, Madden J, Le SQ, Naylor DE, Mucopolysaccharidosis I Intrathecal Research Collaborative. PMID: 26260077; PMCID: PMC4572891.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCTClinical Trials
    73. Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment. Mol Genet Metab. 2015 Sep-Oct; 116(1-2):61-8. Shapiro EG, Nestrasil I, Rudser K, Delaney K, Kovac V, Ahmed A, Yund B, Orchard PJ, Eisengart J, Niklason GR, Raiman J, Mamak E, Cowan MJ, Bailey-Olson M, Harmatz P, Shankar SP, Cagle S, Ali N, Steiner RD, Wozniak J, Lim KO, Whitley CB. PMID: 26095521; PMCID: PMC4561597.
      View in: PubMed   Mentions: 36     Fields:    Translation:HumansCTClinical Trials
    74. Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study. Am J Med Genet A. 2015 Oct; 167A(10):2272-81. Burton BK, Berger KI, Lewis GD, Tarnopolsky M, Treadwell M, Mitchell JJ, Muschol N, Jones SA, Sutton VR, Pastores GM, Lau H, Sparkes R, Genter F, Shaywitz AJ, Harmatz P. PMID: 26069231; PMCID: PMC4744659.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    75. Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase. Mol Genet Metab. 2016 Feb; 117(2):164-71. Smith L, Rhead W, Charrow J, Shankar SP, Bavdekar A, Longo N, Mardach R, Harmatz P, Hangartner T, Lee HM, Crombez E, Pastores GM. PMID: 26043810.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCTClinical Trials
    76. Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome). JIMD Rep. 2015; 18:1-11. Quartel A, Hendriksz CJ, Parini R, Graham S, Lin P, Harmatz P. PMID: 25518809; PMCID: PMC4361922.
      View in: PubMed   Mentions: 21  
    77. Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type II. Mol Genet Metab. 2015 Feb; 114(2):170-7. Yund B, Rudser K, Ahmed A, Kovac V, Nestrasil I, Raiman J, Mamak E, Harmatz P, Steiner R, Lau H, Vekaria P, Wozniak JR, Lim KO, Delaney K, Whitley C, Shapiro EG. PMID: 25541100; PMCID: PMC4312717.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCTClinical Trials
    78. Pulmonary hypertension in well-transfused thalassemia major patients. Blood Cells Mol Dis. 2015 Feb; 54(2):189-94. Meloni A, Detterich J, Pepe A, Harmatz P, Coates TD, Wood JC. PMID: 25488617; PMCID: PMC4297514.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
    79. Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome. Mol Genet Metab. 2015 Feb; 114(2):186-94. Harmatz PR, Mengel KE, Giugliani R, Valayannopoulos V, Lin SP, Parini R, Guffon N, Burton BK, Hendriksz CJ, Mitchell JJ, Martins AM, Jones SA, Guelbert N, Vellodi A, Wijburg FA, Yang K, Slasor P, Decker C. PMID: 25582974.
      View in: PubMed   Mentions: 22     Fields:    Translation:Humans
    80. International guidelines for the management and treatment of Morquio A syndrome. Am J Med Genet A. 2015 Jan; 167A(1):11-25. Hendriksz CJ, Berger KI, Giugliani R, Harmatz P, Kampmann C, Mackenzie WG, Raiman J, Villarreal MS, Savarirayan R. PMID: 25346323; PMCID: PMC4309407.
      View in: PubMed   Mentions: 65     Fields:    Translation:Humans
    81. Morquio A syndrome-associated mutations: a review of alterations in the GALNS gene and a new locus-specific database. Hum Mutat. 2014 Nov; 35(11):1271-9. Morrone A, Caciotti A, Atwood R, Davidson K, Du C, Francis-Lyon P, Harmatz P, Mealiffe M, Mooney S, Oron TR, Ryles A, Zawadzki KA, Miller N. PMID: 25137622; PMCID: PMC4238747.
      View in: PubMed   Mentions: 24     Fields:    Translation:HumansAnimalsPHPublic Health
    82. Mechanisms of plasma non-transferrin bound iron generation: insights from comparing transfused diamond blackfan anaemia with sickle cell and thalassaemia patients. Br J Haematol. 2014 Dec; 167(5):692-6. Porter JB, Walter PB, Neumayr LD, Evans P, Bansal S, Garbowski M, Weyhmiller MG, Harmatz PR, Wood JC, Miller JL, Byrnes C, Weiss G, Seifert M, Grosse R, Grabowski D, Schmidt A, Fischer R, Nielsen P, Niemeyer C, Vichinsky E. PMID: 25209728; PMCID: PMC4577015.
      View in: PubMed   Mentions: 24     Fields:    Translation:HumansCTClinical Trials
    83. Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial. Mol Genet Metab. 2015 Feb; 114(2):178-85. Hendriksz CJ, Giugliani R, Harmatz P, Mengel E, Guffon N, Valayannopoulos V, Parini R, Hughes D, Pastores GM, Lau HA, Al-Sayed MD, Raiman J, STRIVE Investigators, Yang K, Mealiffe M, Haller C. PMID: 25284089.
      View in: PubMed   Mentions: 42     Fields:    Translation:HumansCTClinical Trials
    84. Orthopedic management of the extremities in patients with Morquio A syndrome. J Child Orthop. 2014 Aug; 8(4):295-304. White KK, Jester A, Bache CE, Harmatz PR, Shediac R, Thacker MM, Mackenzie WG. PMID: 25001525; PMCID: PMC4128951.
      View in: PubMed   Mentions: 23  
    85. Quantification of prenatal liver and spleen iron in a sheep model and assessment of iron stores in a human neonate with neonatal hemochromatosis using R2* mapping. Magn Reson Med Sci. 2014; 13(3):167-73. Schoennagel BP, Remus CC, Wedegaertner U, Salzmann I, Grabhorn E, Adam G, Fischer R, Harmatz P, Kooijman H, Yamamura J. PMID: 24990465.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansAnimals
    86. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014 Nov; 37(6):979-90. Hendriksz CJ, Burton B, Fleming TR, Harmatz P, Hughes D, Jones SA, Lin SP, Mengel E, Scarpa M, Valayannopoulos V, Giugliani R, STRIVE Investigators, Slasor P, Lounsbury D, Dummer W. PMID: 24810369; PMCID: PMC4206772.
      View in: PubMed   Mentions: 99     Fields:    Translation:HumansCTClinical Trials
    87. Natural history and galsulfase treatment in mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome)--10-year follow-up of patients who previously participated in an MPS VI Survey Study. Am J Med Genet A. 2014 Aug; 164A(8):1953-64. Giugliani R, Lampe C, Guffon N, Ketteridge D, Leão-Teles E, Wraith JE, Jones SA, Piscia-Nichols C, Lin P, Quartel A, Harmatz P. PMID: 24764221; PMCID: PMC4107078.
      View in: PubMed   Mentions: 34     Fields:    Translation:Humans
    88. Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI. J Inherit Metab Dis. 2014 Mar; 37(2):277-87. Harmatz PR, Garcia P, Guffon N, Randolph LM, Shediac R, Braunlin E, Lachman RS, Decker C. PMID: 24108527; PMCID: PMC3976509.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCTClinical Trials
    89. Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure. Orphanet J Rare Dis. 2013 Oct 03; 8:155. Langereis EJ, Borgo A, Crushell E, Harmatz PR, van Hasselt PM, Jones SA, Kelly PM, Lampe C, van der Lee JH, Odent T, Sakkers R, Scarpa M, Schafroth MU, Struijs PA, Valayannopoulos V, White KK, Wijburg FA. PMID: 24088413; PMCID: PMC3852175.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    90. Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients. Pediatr Res. 2013 Dec; 74(6):712-20. Vera M, Le S, Kan SH, Garban H, Naylor D, Mlikotic A, Kaitila I, Harmatz P, Chen A, Dickson P. PMID: 24002329; PMCID: PMC3855632.
      View in: PubMed   Mentions: 12     Fields:    Translation:Humans
    91. Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT. PLoS One. 2013; 8(6):e67052. Banugaria SG, Prater SN, Patel TT, Dearmey SM, Milleson C, Sheets KB, Bali DS, Rehder CW, Raiman JA, Wang RA, Labarthe F, Charrow J, Harmatz P, Chakraborty P, Rosenberg AS, Kishnani PS. PMID: 23825616; PMCID: PMC3692419.
      View in: PubMed   Mentions: 53     Fields:    Translation:Humans
    92. Development of a Scoring System to Evaluate the Severity of Craniocervical Spinal Cord Compression in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome). JIMD Rep. 2013; 11:65-72. Möllmann C, Lampe CG, Müller-Forell W, Scarpa M, Harmatz P, Schwarz M, Beck M, Lampe C. PMID: 23580366; PMCID: PMC3755552.
      View in: PubMed   Mentions: 13  
    93. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA. Mol Genet Metab. 2013 Sep-Oct; 110(1-2):54-64. Hendriksz CJ, Harmatz P, Beck M, Jones S, Wood T, Lachman R, Gravance CG, Orii T, Tomatsu S. PMID: 23665161; PMCID: PMC3755102.
      View in: PubMed   Mentions: 78     Fields:    Translation:HumansCells
    94. The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS). Mol Genet Metab. 2013 May; 109(1):41-8. Jones SA, Parini R, Harmatz P, Giugliani R, Fang J, Mendelsohn NJ, HOS Natural History Working Group on behalf of HOS Investigators. PMID: 23537841.
      View in: PubMed   Mentions: 36     Fields:    Translation:Humans
    95. Craniocervical decompression in patients with mucopolysaccharidosis VI: development of a scoring system to determine indication and outcome of surgery. J Inherit Metab Dis. 2013 Nov; 36(6):1005-13. Lampe C, Lampe C, Schwarz M, Müller-Forell W, Harmatz P, Mengel E. PMID: 23408180.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    96. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. Mol Genet Metab. 2013 May; 109(1):54-61. Harmatz P, Mengel KE, Giugliani R, Valayannopoulos V, Lin SP, Parini R, Guffon N, Burton BK, Hendriksz CJ, Mitchell J, Martins A, Jones S, Guelbert N, Vellodi A, Hollak C, Slasor P, Decker C. PMID: 23452954.
      View in: PubMed   Mentions: 64     Fields:    Translation:Humans
    97. Spinal involvement in mucopolysaccharidosis IVA (Morquio-Brailsford or Morquio A syndrome): presentation, diagnosis and management. J Inherit Metab Dis. 2013 Mar; 36(2):339-55. Solanki GA, Martin KW, Theroux MC, Lampe C, White KK, Shediac R, Lampe CG, Beck M, Mackenzie WG, Hendriksz CJ, Harmatz PR. PMID: 23385297; PMCID: PMC3590412.
      View in: PubMed   Mentions: 42     Fields:    Translation:Humans
    98. Diagnosing mucopolysaccharidosis IVA. J Inherit Metab Dis. 2013 Mar; 36(2):293-307. Wood TC, Harvey K, Beck M, Burin MG, Chien YH, Church HJ, D'Almeida V, van Diggelen OP, Fietz M, Giugliani R, Harmatz P, Hawley SM, Hwu WL, Ketteridge D, Lukacs Z, Miller N, Pasquali M, Schenone A, Thompson JN, Tylee K, Yu C, Hendriksz CJ. PMID: 23371450; PMCID: PMC3590423.
      View in: PubMed   Mentions: 49     Fields:    Translation:HumansCells
    99. Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase. Am J Hematol. 2013 Mar; 88(3):172-8. Zimran A, Pastores GM, Tylki-Szymanska A, Hughes DA, Elstein D, Mardach R, Eng C, Smith L, Heisel-Kurth M, Charrow J, Harmatz P, Fernhoff P, Rhead W, Longo N, Giraldo P, Ruiz JA, Zahrieh D, Crombez E, Grabowski GA. PMID: 23339116; PMCID: PMC3586535.
      View in: PubMed   Mentions: 16     Fields:    Translation:HumansCTClinical Trials
    100. Exercise performance in thalassemia major: correlation with cardiac iron burden. Am J Hematol. 2013 Mar; 88(3):193-7. Sohn EY, Kato R, Noetzli LJ, Gera A, Coates T, Harmatz P, Keens TG, Wood JC. PMID: 23339082.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    101. Increased leucocyte apoptosis in transfused β-thalassaemia patients. Br J Haematol. 2013 Feb; 160(3):399-403. Walter PB, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Grady RW, Vichinsky E, Olivieri N, Trachtenberg F, Alberti D, Fung E, Ames B, Higa A, Harmatz P, Thalassemia Clinical Research Network. PMID: 23216540; PMCID: PMC4597914.
      View in: PubMed   Mentions: 3     Fields:    Translation:HumansCells
    102. Anaesthesia and airway management in mucopolysaccharidosis. J Inherit Metab Dis. 2013 Mar; 36(2):211-9. Walker R, Belani KG, Braunlin EA, Bruce IA, Hack H, Harmatz PR, Jones S, Rowe R, Solanki GA, Valdemarsson B. PMID: 23197104; PMCID: PMC3590422.
      View in: PubMed   Mentions: 43     Fields:    Translation:Humans
    103. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood Cells Mol Dis. 2013 Feb; 50(2):99-104. Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. PMID: 23151373; PMCID: PMC3592978.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCTClinical Trials
    104. A multinational, multidisciplinary consensus for the diagnosis and management of spinal cord compression among patients with mucopolysaccharidosis VI. Mol Genet Metab. 2012 Sep; 107(1-2):15-24. Solanki GA, Alden TD, Burton BK, Giugliani R, Horovitz DD, Jones SA, Lampe C, Martin KW, Ryan ME, Schaefer MK, Siddiqui A, White KK, Harmatz P. PMID: 22938833.
      View in: PubMed   Mentions: 12     Fields:    Translation:Humans
    105. Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy. J Inherit Metab Dis. 2013 Mar; 36(2):385-94. Braunlin E, Rosenfeld H, Kampmann C, Johnson J, Beck M, Giugliani R, Guffon N, Ketteridge D, Sá Miranda CM, Scarpa M, Schwartz IV, Leão Teles E, Wraith JE, Barrios P, Dias da Silva E, Kurio G, Richardson M, Gildengorin G, Hopwood JJ, Imperiale M, Schatz A, Decker C, Harmatz P, MPS VI Study Group. PMID: 22669363; PMCID: PMC3590402.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    106. Expert recommendations for the laboratory diagnosis of MPS VI. Mol Genet Metab. 2012 May; 106(1):73-82. Wood T, Bodamer OA, Burin MG, D'Almeida V, Fietz M, Giugliani R, Hawley SM, Hendriksz CJ, Hwu WL, Ketteridge D, Lukacs Z, Mendelsohn NJ, Miller N, Pasquali M, Schenone A, Schoonderwoerd K, Winchester B, Harmatz P. PMID: 22405600.
      View in: PubMed   Mentions: 20     Fields:    Translation:Humans
    107. A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. Blood. 2012 Apr 05; 119(14):3263-8. Neufeld EJ, Galanello R, Viprakasit V, Aydinok Y, Piga A, Harmatz P, Forni GL, Shah FT, Grace RF, Porter JB, Wood JC, Peppe J, Jones A, Rienhoff HY. PMID: 22251482; PMCID: PMC3321852.
      View in: PubMed   Mentions: 17     Fields:    Translation:HumansCTClinical Trials
    108. Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP). J Inherit Metab Dis. 2013 Mar; 36(2):373-84. Hendriksz CJ, Giugliani R, Harmatz P, Lampe C, Martins AM, Pastores GM, Steiner RD, Leão Teles E, Valayannopoulos V, CSP Study Group. PMID: 22127392.
      View in: PubMed   Mentions: 30     Fields:    Translation:Humans
    109. Electrocardiographic consequences of cardiac iron overload in thalassemia major. Am J Hematol. 2012 Feb; 87(2):139-44. Detterich J, Noetzli L, Dorey F, Bar-Cohen Y, Harmatz P, Coates T, Wood J. PMID: 22052662; PMCID: PMC3306475.
      View in: PubMed   Mentions: 25     Fields:    Translation:Humans
    110. The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus. Eur J Pediatr. 2012 Jan; 171(1):181-8. Muenzer J, Bodamer O, Burton B, Clarke L, Frenking GS, Giugliani R, Jones S, Rojas MV, Scarpa M, Beck M, Harmatz P. PMID: 22037758; PMCID: PMC3249184.
      View in: PubMed   Mentions: 44     Fields:    Translation:Humans
    111. Iron chelation therapy in thalassemia major: a systematic review with meta-analyses of 1520 patients included on randomized clinical trials. Blood Cells Mol Dis. 2011 Oct 15; 47(3):166-75. Maggio A, Filosa A, Vitrano A, Aloj G, Kattamis A, Ceci A, Fucharoen S, Cianciulli P, Grady RW, Prossomariti L, Porter JB, Iacono A, Cappellini MD, Bonifazi F, Cassarà F, Harmatz P, Wood J, Gluud C. PMID: 21843958.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    112. Pulmonary function in thalassaemia major and its correlation with body iron stores. Br J Haematol. 2011 Oct; 155(1):102-5. Sohn EY, Noetzli LJ, Gera A, Kato R, Coates TD, Harmatz P, Keens TG, Wood JC. PMID: 21810090; PMCID: PMC3169737.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    113. Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management. J Inherit Metab Dis. 2011 Dec; 34(6):1183-97. Braunlin EA, Harmatz PR, Scarpa M, Furlanetto B, Kampmann C, Loehr JP, Ponder KP, Roberts WC, Rosenfeld HM, Giugliani R. PMID: 21744090; PMCID: PMC3228957.
      View in: PubMed   Mentions: 113     Fields:    Translation:Humans
    114. Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial. Haematologica. 2011 Jul; 96(7):1055-8. Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. PMID: 21393329; PMCID: PMC3128226.
      View in: PubMed   Mentions: 18     Fields:    Translation:HumansCTClinical Trials
    115. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genet Med. 2011 Feb; 13(2):95-101. Muenzer J, Beck M, Eng CM, Giugliani R, Harmatz P, Martin R, Ramaswami U, Vellodi A, Wraith JE, Cleary M, Gucsavas-Calikoglu M, Puga AC, Shinawi M, Ulbrich B, Vijayaraghavan S, Wendt S, Conway AM, Rossi A, Whiteman DA, Kimura A. PMID: 21150784.
      View in: PubMed   Mentions: 97     Fields:    Translation:Humans
    116. A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload. Haematologica. 2011 Apr; 96(4):521-5. Rienhoff HY, Viprakasit V, Tay L, Harmatz P, Vichinsky E, Chirnomas D, Kwiatkowski JL, Tapper A, Kramer W, Porter JB, Neufeld EJ. PMID: 21173101; PMCID: PMC3069228.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCTClinical Trials
    117. Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey. Genet Med. 2010 Dec; 12(12):816-22. Mendelsohn NJ, Harmatz P, Bodamer O, Burton BK, Giugliani R, Jones SA, Lampe C, Malm G, Steiner RD, Parini R, Hunter Outcome Survey Investigators. PMID: 21045710.
      View in: PubMed   Mentions: 36     Fields:    Translation:Humans
    118. Follow-up report on the 2-year cardiac data from a deferasirox monotherapy trial. Am J Hematol. 2010 Oct; 85(10):818-9. Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. PMID: 20799360.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansCTClinical Trials
    119. Diagnosis and management of ophthalmological features in patients with mucopolysaccharidosis. Br J Ophthalmol. 2011 May; 95(5):613-9. Ferrari S, Ponzin D, Ashworth JL, Fahnehjelm KT, Summers CG, Harmatz PR, Scarpa M. PMID: 20852315.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    120. Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures. Turk J Pediatr. 2010 Sep-Oct; 52(5):443-9. Harmatz P. PMID: 21434527.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    121. Management of chronic viral hepatitis in patients with thalassemia: recommendations from an international panel. Blood. 2010 Oct 21; 116(16):2875-83. Di Marco V, Capra M, Angelucci E, Borgna-Pignatti C, Telfer P, Harmatz P, Kattamis A, Prossamariti L, Filosa A, Rund D, Gamberini MR, Cianciulli P, De Montalembert M, Gagliardotto F, Foster G, Grangè JD, Cassarà F, Iacono A, Cappellini MD, Brittenham GM, Prati D, Pietrangelo A, Craxì A, Maggio A, Italian Society for the Study of Thalassemia and Haemoglobinopathies, Italian Association for the Study of the Liver. PMID: 20551378.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    122. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood. 2010 Jul 29; 116(4):537-43. Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD. PMID: 20421452.
      View in: PubMed   Mentions: 48     Fields:    Translation:HumansCTClinical Trials
    123. Mucopolysaccharidosis VI. Orphanet J Rare Dis. 2010 Apr 12; 5:5. Valayannopoulos V, Nicely H, Harmatz P, Turbeville S. PMID: 20385007; PMCID: PMC2873242.
      View in: PubMed   Mentions: 117     Fields:    Translation:Humans
    124. Elevated exhaled carbon monoxide concentration in hemoglobinopathies and its relation to red blood cell transfusion therapy. Pediatr Hematol Oncol. 2010 Mar; 27(2):112-21. James EB, Vreman HJ, Wong RJ, Stevenson DK, Vichinsky E, Schumacher L, Hall JY, Simon J, Golden DW, Harmatz P. PMID: 20201692.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    125. Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. J Inherit Metab Dis. 2010 Feb; 33(1):51-60. Harmatz P, Yu ZF, Giugliani R, Schwartz IV, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Swiedler SJ, Hardy K, Berger KI, Decker C. PMID: 20140523; PMCID: PMC2828556.
      View in: PubMed   Mentions: 30     Fields:    Translation:Humans
    126. Assessment of cardiac iron by MRI susceptometry and R2* in patients with thalassemia. Magn Reson Imaging. 2010 Apr; 28(3):363-71. Wang ZJ, Fischer R, Chu Z, Mahoney DH, Mueller BU, Muthupillai R, James EB, Krishnamurthy R, Chung T, Padua E, Vichinsky E, Harmatz P. PMID: 20061110; PMCID: PMC2839041.
      View in: PubMed   Mentions: 9     Fields:    Translation:Humans
    127. Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. J Pediatr Rehabil Med. 2010; 3(2):89-100. Decker C, Yu ZF, Giugliani R, Schwartz IV, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Swiedler SJ, Conrad S, Harmatz P. PMID: 20634905; PMCID: PMC2904323.
      View in: PubMed   Mentions: 32     Fields:    
    128. Orthopedic management of mucopolysaccharide disease. J Pediatr Rehabil Med. 2010; 3(1):47-56. White KK, Harmatz P. PMID: 21791829.
      View in: PubMed   Mentions: 21     Fields:    
    129. Bone density assessment in patients with mucopolysaccharidosis: A preliminary report from patients with MPS II and VI. J Pediatr Rehabil Med. 2010; 3(1):13-23. Fung EB, Johnson JA, Madden J, Kim T, Harmatz P. PMID: 20617160; PMCID: PMC2898152.
      View in: PubMed   Mentions: 24     Fields:    
    130. Iron metabolism and iron chelation in sickle cell disease. Acta Haematol. 2009; 122(2-3):174-83. Walter PB, Harmatz P, Vichinsky E. PMID: 19907155.
      View in: PubMed   Mentions: 21     Fields:    Translation:Humans
    131. Multidisciplinary management of Hunter syndrome. Pediatrics. 2009 Dec; 124(6):e1228-39. Muenzer J, Beck M, Eng CM, Escolar ML, Giugliani R, Guffon NH, Harmatz P, Kamin W, Kampmann C, Koseoglu ST, Link B, Martin RA, Molter DW, Muñoz Rojas MV, Ogilvie JW, Parini R, Ramaswami U, Scarpa M, Schwartz IV, Wood RE, Wraith E. PMID: 19901005.
      View in: PubMed   Mentions: 72     Fields:    Translation:Humans
    132. Patterns of hepatic iron distribution in patients with chronically transfused thalassemia and sickle cell disease. Am J Hematol. 2009 Aug; 84(8):480-3. Ghugre NR, Gonzalez-Gomez I, Butensky E, Noetzli L, Fischer R, Williams R, Harmatz P, Coates TD, Wood JC. PMID: 19536851; PMCID: PMC2884400.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCells
    133. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. J Pediatr. 2009 May; 154(5):700-7. Trefz FK, Burton BK, Longo N, Casanova MM, Gruskin DJ, Dorenbaum A, Kakkis ED, Crombez EA, Grange DK, Harmatz P, Lipson MH, Milanowski A, Randolph LM, Vockley J, Whitley CB, Wolff JA, Bebchuk J, Christ-Schmidt H, Hennermann JB, Sapropterin Study Group. PMID: 19261295.
      View in: PubMed   Mentions: 64     Fields:    Translation:HumansCTClinical Trials
    134. Altered iron metabolism in children with human immunodeficiency virus disease. Pediatr Hematol Oncol. 2009 Mar; 26(2):69-84. Butensky James E, Harmatz P, Lee M, Kennedy C, Petru A, Wara D, Miaskowski C. PMID: 19322737.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    135. Non-invasive assessment of tissue iron overload. Hematology Am Soc Hematol Educ Program. 2009; 215-21. Fischer R, Harmatz PR. PMID: 20008201.
      View in: PubMed   Mentions: 32     Fields:    Translation:Humans
    136. Folate concentrations in pediatric patients with newly diagnosed inflammatory bowel disease. Am J Clin Nutr. 2009 Feb; 89(2):545-50. Heyman MB, Garnett EA, Shaikh N, Huen K, Jose FA, Harmatz P, Winter HS, Baldassano RN, Cohen SA, Gold BD, Kirschner BS, Ferry GD, Stege E, Holland N. PMID: 19116333; PMCID: PMC2647761.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    137. Italian Society of Hematology guidelines for thalassemia and non-invasive iron measurements. Haematologica. 2009 Feb; 94(2):294-5; author reply 295-6. Nielsen P, Engelhardt R, Grosse R, Janka G, Harmatz P, Fischer R. PMID: 19109216; PMCID: PMC2635390.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    138. Vitamin C treatment reduces elevated C-reactive protein. Free Radic Biol Med. 2009 Jan 01; 46(1):70-7. Block G, Jensen CD, Dalvi TB, Norkus EP, Hudes M, Crawford PB, Holland N, Fung EB, Schumacher L, Harmatz P. PMID: 18952164; PMCID: PMC2631578.
      View in: PubMed   Mentions: 41     Fields:    Translation:HumansCTClinical Trials
    139. Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia. Haematologica. 2008 Aug; 93(8):1247-51. Harmatz P, Jonas MM, Kwiatkowski JL, Wright EC, Fischer R, Vichinsky E, Giardina PJ, Neufeld EJ, Porter J, Olivieri N, Thalassemia Clinical Research Network. PMID: 18556414.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCellsCTClinical Trials
    140. Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions. Transfusion. 2008 Sep; 48(9):1971-80. Fung EB, Harmatz PR, Milet M, Balasa V, Ballas SK, Casella JF, Hilliard L, Kutlar A, McClain KL, Olivieri NF, Porter JB, Vichinsky EP, Multi-Center Iron Overload Research Group. PMID: 18513257; PMCID: PMC4613769.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    141. Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab. 2008 Aug; 94(4):469-475. Harmatz P, Giugliani R, D Schwartz IV, Guffon N, Teles EL, Miranda MCS, Wraith JE, Beck M, Arash L, Scarpa M, Ketteridge D, Hopwood JJ, Plecko B, Steiner R, Whitley CB, Kaplan P, Yu ZF, Swiedler SJ, Decker C, MPS VI Study Group. PMID: 18502162.
      View in: PubMed   Mentions: 94     Fields:    Translation:HumansCTClinical Trials
    142. Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial. Haematologica. 2008 Jun; 93(6):817-25. Walter PB, Macklin EA, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Vichinsky E, Olivieri N, Alberti D, Holland J, Harmatz P, Thalassemia Clinical Research Network. PMID: 18469351.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCellsCTClinical Trials
    143. Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapy. Int Ophthalmol. 2009 Aug; 29(4):267-9. Koseoglu ST, Harmatz P, Turbeville S, Nicely H. PMID: 18418554; PMCID: PMC2714452.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    144. The effect of vitamins C and E on biomarkers of oxidative stress depends on baseline level. Free Radic Biol Med. 2008 Aug 15; 45(4):377-84. Block G, Jensen CD, Morrow JD, Holland N, Norkus EP, Milne GL, Hudes M, Dalvi TB, Crawford PB, Fung EB, Schumacher L, Harmatz P. PMID: 18455517; PMCID: PMC2750000.
      View in: PubMed   Mentions: 42     Fields:    Translation:Humans
    145. Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia. Bone. 2008 Jul; 43(1):162-168. Fung EB, Harmatz PR, Milet M, Coates TD, Thompson AA, Ranalli M, Mignaca R, Scher C, Giardina P, Robertson S, Neumayr L, Vichinsky EP, Multi-Center Iron Overload Study Group. PMID: 18430624; PMCID: PMC2500183.
      View in: PubMed   Mentions: 29     Fields:    Translation:Humans
    146. Reduced intracellular T-helper 1 interferon-gamma in blood of newly diagnosed children with Crohn's disease and age-related changes in Th1/Th2 cytokine profiles. Pediatr Res. 2008 Mar; 63(3):257-62. Holland N, Dong J, Garnett E, Shaikh N, Huen K, Harmatz P, Olive A, Winter HS, Gold BD, Cohen SA, Baldassano RN, Kirschner BS, Heyman MB. PMID: 18287963; PMCID: PMC3252046.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCells
    147. Entering a new treatment age for mucopolysaccharidosis VI disease: a search for better markers of disease progression and response to treatment. J Pediatr (Rio J). 2008 Mar-Apr; 84(2):103-6. Harmatz P. PMID: 18372931.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    148. Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics. 2008 Feb; 121(2):e377-86. Martin R, Beck M, Eng C, Giugliani R, Harmatz P, Muñoz V, Muenzer J. PMID: 18245410.
      View in: PubMed   Mentions: 119     Fields:    Translation:Humans
    149. Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies. Pediatr Blood Cancer. 2008 Feb; 50(2):319-24. Jastaniah W, Harmatz P, Pakbaz Z, Fischer R, Vichinsky E, Walters MC. PMID: 17557314.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    150. Pharmacokinetics of sapropterin in patients with phenylketonuria. Clin Pharmacokinet. 2008; 47(12):817-25. Feillet F, Clarke L, Meli C, Lipson M, Morris AA, Harmatz P, Mould DR, Green B, Dorenbaum A, Giovannini M, Foehr E, Sapropterin Research Group. PMID: 19026037.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    151. Mutational analysis of 105 mucopolysaccharidosis type VI patients. Hum Mutat. 2007 Sep; 28(9):897-903. Karageorgos L, Brooks DA, Pollard A, Melville EL, Hein LK, Clements PR, Ketteridge D, Swiedler SJ, Beck M, Giugliani R, Harmatz P, Wraith JE, Guffon N, Leão Teles E, Sá Miranda MC, Hopwood JJ. PMID: 17458871.
      View in: PubMed   Mentions: 49     Fields:    Translation:HumansCells
    152. Serum ferritin underestimates liver iron concentration in transfusion independent thalassemia patients as compared to regularly transfused thalassemia and sickle cell patients. Pediatr Blood Cancer. 2007 Sep; 49(3):329-32. Pakbaz Z, Fischer R, Fung E, Nielsen P, Harmatz P, Vichinsky E. PMID: 17554789.
      View in: PubMed   Mentions: 32     Fields:    Translation:Humans
    153. Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator. Br J Haematol. 2007 Aug; 138(3):374-81. Harmatz P, Grady RW, Dragsten P, Vichinsky E, Giardina P, Madden J, Jeng M, Miller B, Hanson G, Hedlund B. PMID: 17614825.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCTClinical Trials
    154. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007 Aug; 120(2):405-18. Giugliani R, Harmatz P, Wraith JE. PMID: 17671068.
      View in: PubMed   Mentions: 73     Fields:    Translation:Humans
    155. Iron homeostasis during transfusional iron overload in beta-thalassemia and sickle cell disease: changes in iron regulatory protein, hepcidin, and ferritin expression. Pediatr Hematol Oncol. 2007 Jun; 24(4):237-43. Jenkins ZA, Hagar W, Bowlus CL, Johansson HE, Harmatz P, Vichinsky EP, Theil EC. PMID: 17613866.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    156. Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload. Am J Hematol. 2007 Apr; 82(4):255-65. Fung EB, Harmatz P, Milet M, Ballas SK, De Castro L, Hagar W, Owen W, Olivieri N, Smith-Whitley K, Darbari D, Wang W, Vichinsky E, Multi-Center Study of Iron Overload Research Group. PMID: 17094096.
      View in: PubMed   Mentions: 56     Fields:    Translation:Humans
    157. Cytogenetic damage in blood lymphocytes and exfoliated epithelial cells of children with inflammatory bowel disease. Pediatr Res. 2007 Feb; 61(2):209-14. Holland N, Harmatz P, Golden D, Hubbard A, Wu YY, Bae J, Chen C, Huen K, Heyman MB. PMID: 17237724.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCells
    158. Caregiving time in sickle cell disease: psychological effects in maternal caregivers. Pediatr Blood Cancer. 2007 Jan; 48(1):64-71. Moskowitz JT, Butensky E, Harmatz P, Vichinsky E, Heyman MB, Acree M, Wrubel J, Wilson L, Folkman S. PMID: 16622841.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCTClinical Trials
    159. Mutational analysis of mucopolysaccharidosis type VI patients undergoing a phase II trial of enzyme replacement therapy. Mol Genet Metab. 2007 Feb; 90(2):164-70. Karageorgos L, Brooks DA, Harmatz P, Ketteridge D, Pollard A, Melville EL, Parkinson-Lawrence E, Clements PR, Hopwood JJ. PMID: 17161971.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCTClinical Trials
    160. Increased prevalence of iron-overload associated endocrinopathy in thalassaemia versus sickle-cell disease. Br J Haematol. 2006 Nov; 135(4):574-82. Fung EB, Harmatz PR, Lee PD, Milet M, Bellevue R, Jeng MR, Kalinyak KA, Hudes M, Bhatia S, Vichinsky EP, Multi-Centre Study of Iron Overload Research Group. PMID: 17054676.
      View in: PubMed   Mentions: 60     Fields:    Translation:Humans
    161. Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease. Br J Haematol. 2006 Oct; 135(2):254-63. Walter PB, Fung EB, Killilea DW, Jiang Q, Hudes M, Madden J, Porter J, Evans P, Vichinsky E, Harmatz P. PMID: 17010049; PMCID: PMC2185791.
      View in: PubMed   Mentions: 92     Fields:    Translation:Humans
    162. Does liver biopsy overestimate liver iron concentration? Blood. 2006 Sep 01; 108(5):1775-6; author reply 1776. Fischer R, Harmatz P, Nielsen P. PMID: 16926297.
      View in: PubMed   Mentions: 7     Fields:    Translation:Humans
    163. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med. 2006 Aug; 8(8):465-73. Muenzer J, Wraith JE, Beck M, Giugliani R, Harmatz P, Eng CM, Vellodi A, Martin R, Ramaswami U, Gucsavas-Calikoglu M, Vijayaraghavan S, Wendt S, Wendt S, Puga AC, Puga A, Ulbrich B, Shinawi M, Cleary M, Piper D, Conway AM, Conway AM, Kimura A. PMID: 16912578.
      View in: PubMed   Mentions: 233     Fields:    Translation:HumansCTClinical Trials
    164. Does liver biopsy overestimate liver iron concentration? Blood. 2006 Jul 15; 108(2):778. Fischer R, Harmatz P, Nielsen P. PMID: 16822911.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    165. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study. J Pediatr. 2006 Apr; 148(4):533-539. Harmatz P, Giugliani R, Schwartz I, Guffon N, Teles EL, Miranda MC, Wraith JE, Beck M, Arash L, Scarpa M, Yu ZF, Wittes J, Berger KI, Newman MS, Lowe AM, Kakkis E, Swiedler SJ, MPS VI Phase 3 Study Group. PMID: 16647419.
      View in: PubMed   Mentions: 125     Fields:    Translation:HumansCTClinical Trials
    166. Comparison of organ dysfunction in transfused patients with SCD or beta thalassemia. Am J Hematol. 2005 Sep; 80(1):70-4. Vichinsky E, Butensky E, Fung E, Hudes M, Theil E, Ferrell L, Williams R, Louie L, Lee PD, Harmatz P. PMID: 16138345.
      View in: PubMed   Mentions: 49     Fields:    Translation:Humans
    167. The economic burden of home care for children with HIV and other chronic illnesses. Am J Public Health. 2005 Aug; 95(8):1445-52. Wilson LS, Moskowitz JT, Acree M, Heyman MB, Harmatz P, Ferrando SJ, Folkman S. PMID: 15985648; PMCID: PMC1449379.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    168. Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase. Pediatrics. 2005 Jun; 115(6):e681-9. Harmatz P, Ketteridge D, Giugliani R, Guffon N, Teles EL, Miranda MC, Yu ZF, Swiedler SJ, Hopwood JJ, MPS VI Study Group. PMID: 15930196.
      View in: PubMed   Mentions: 73     Fields:    Translation:HumansCTClinical Trials
    169. Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet A. 2005 Apr 15; 134A(2):144-50. Swiedler SJ, Beck M, Bajbouj M, Giugliani R, Schwartz I, Harmatz P, Wraith JE, Roberts J, Ketteridge D, Hopwood JJ, Guffon N, Sá Miranda MC, Teles EL, Berger KI, Piscia-Nichols C. PMID: 15690405.
      View in: PubMed   Mentions: 51     Fields:    Translation:Humans
    170. Pharmacokinetic profile of recombinant human N-acetylgalactosamine 4-sulphatase enzyme replacement therapy in patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): a phase I/II study. Acta Paediatr Suppl. 2005 Mar; 94(447):61-8; discussion 57. Harmatz P, Kramer WG, Hopwood JJ, Simon J, Butensky E, Swiedler SJ, Mucopolysaccharidosis VI Study Group. PMID: 15895715.
      View in: PubMed   Mentions: 20     Fields:    Translation:HumansCTClinical Trials
    171. A simple model to assess and improve adherence to iron chelation therapy with deferoxamine in patients with thalassemia. Ann N Y Acad Sci. 2005; 1054:486-91. Pakbaz Z, Fischer R, Treadwell M, Yamashita R, Fung EB, Calvelli L, Quirolo K, Foote D, Harmatz P, Vichinsky EP. PMID: 16339703.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    172. Monitoring long-term efficacy of iron chelation treatment with biomagnetic liver susceptometry. Ann N Y Acad Sci. 2005; 1054:350-7. Fischer R, Piga A, Harmatz P, Nielsen P. PMID: 16339683.
      View in: PubMed   Mentions: 26     Fields:    Translation:Humans
    173. Treatment of hepatitis C virus infection in thalassemia. Ann N Y Acad Sci. 2005; 1054:290-9. Butensky E, Pakbaz Z, Foote D, Walters MC, Vichinsky EP, Harmatz P. PMID: 16339677.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    174. Variability in hepatic iron concentration in percutaneous needle biopsy specimens from patients with transfusional hemosiderosis. Am J Clin Pathol. 2005 Jan; 123(1):146-52. Butensky E, Fischer R, Hudes M, Schumacher L, Williams R, Moyer TP, Vichinsky E, Harmatz P. PMID: 15762291.
      View in: PubMed   Mentions: 19     Fields:    Translation:Humans
    175. Interaction of artificial metallic objects with biosusceptometric measurements. Neurol Clin Neurophysiol. 2004 Nov 30; 2004:32. Engelhardt R, Fung EB, Kelly P, Biehl TR, Pakbaz Z, Nielsen P, Harmatz P, Fischer R. PMID: 16012652.
      View in: PubMed   Mentions:    Fields:    Translation:Humans
    176. The new SQUID biosusceptometer at Oakland: first year of experience. Neurol Clin Neurophysiol. 2004 Nov 30; 2004:5. Fung EB, Fischer R, Pakbaz Z, Fagaly RL, Vichinsky E, Starr TN, Ewing T, Paulson DN, Hassenzahl WV, Harmatz P. PMID: 16012600.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    177. Potential mechanisms for altered iron metabolism in human immunodeficiency virus disease. J Assoc Nurses AIDS Care. 2004 Nov-Dec; 15(6):31-45. Butensky E, Kennedy CM, Lee MM, Harmatz P, Miaskowski C. PMID: 15538015.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    178. Economic and psychologic costs for maternal caregivers of gastrostomy-dependent children. J Pediatr. 2004 Oct; 145(4):511-6. Heyman MB, Harmatz P, Acree M, Wilson L, Moskowitz JT, Ferrando S, Folkman S. PMID: 15480377.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    179. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr. 2004 May; 144(5):574-80. Harmatz P, Whitley CB, Waber L, Pais R, Steiner R, Plecko B, Kaplan P, Simon J, Butensky E, Hopwood JJ. PMID: 15126989.
      View in: PubMed   Mentions: 94     Fields:    Translation:HumansCTClinical Trials
    180. Mutational analysis of mucopolysaccharidosis type VI patients undergoing a trial of enzyme replacement therapy. Hum Mutat. 2004 Mar; 23(3):229-33. Karageorgos L, Harmatz P, Simon J, Pollard A, Clements PR, Brooks DA, Hopwood JJ. PMID: 14974081.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansAnimalsCellsCTClinical Trials
    181. Correlation of abnormal intracranial vessel velocity, measured by transcranial Doppler ultrasonography, with abnormal conjunctival vessel velocity, measured by computer-assisted intravital microscopy, in sickle cell disease. Blood. 2001 Jun 01; 97(11):3401-4. Cheung AT, Harmatz P, Wun T, Chen PC, Larkin EC, Adams RJ, Vichinsky EP. PMID: 11369629.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    182. Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy. Blood. 2000 Jul 01; 96(1):76-9. Harmatz P, Butensky E, Quirolo K, Williams R, Ferrell L, Moyer T, Golden D, Neumayr L, Vichinsky E. PMID: 10891433.
      View in: PubMed   Mentions: 63     Fields:    Translation:HumansCTClinical Trials
    183. Validation of the Natus CO-Stat End Tidal Breath Analyzer in children and adults. J Clin Monit Comput. 1999 Dec; 15(7-8):421-7. Vreman HJ, Wong RJ, Harmatz P, Fanaroff AA, Berman B, Stevenson DK. PMID: 12578038.
      View in: PubMed   Mentions: 6     Fields:    Translation:HumansCTClinical Trials
    184. Effects of red blood cell transfusion on resting energy expenditure in adolescents with sickle cell anemia. J Pediatr Gastroenterol Nutr. 1999 Aug; 29(2):127-31. Harmatz P, Heyman MB, Cunningham J, Lee PD, Styles L, Quirolo K, Kopp-Hoolihan L, Ghiron J, Hintz RL, Vichinsky E. PMID: 10435647.
      View in: PubMed   Mentions: 5     Fields:    Translation:HumansCTClinical Trials
    185. Polyamines in human and rat milk influence intestinal cell growth in vitro. J Pediatr Gastroenterol Nutr. 1998 Sep; 27(3):281-6. Capano G, Bloch KJ, Carter EA, Dascoli JA, Schoenfeld D, Harmatz PR. PMID: 9740197.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimalsCells
    186. Low ceruloplasmin levels during recovery from major burn injury: influence of open wound size and copper supplementation. Nutrition. 1996 Feb; 12(2):83-8. Cunningham JJ, Lydon MK, Emerson R, Harmatz PR. PMID: 8724377.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    187. Elevated plasma ceruloplasmin in insulin-dependent diabetes mellitus: evidence for increased oxidative stress as a variable complication. Metabolism. 1995 Aug; 44(8):996-9. Cunningham J, Leffell M, Mearkle P, Harmatz P. PMID: 7637657.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    188. Influence of the polyamine, spermidine, on intestinal maturation and dietary antigen uptake in the neonatal rat. J Pediatr Gastroenterol Nutr. 1994 Jul; 19(1):34-42. Capano G, Bloch KJ, Schiffrin EJ, Dascoli JA, Israel EJ, Harmatz PR. PMID: 7965474.
      View in: PubMed   Mentions: 5     Fields:    Translation:Animals
    189. Ontogeny of Ia messenger RNA in the mouse small intestinal epithelium is modulated by age of weaning and diet. Gastroenterology. 1993 Oct; 105(4):974-80. Sanderson IR, Ouellette AJ, Carter EA, Harmatz PR. PMID: 8405883.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCells
    190. Burn severity, copper dose, and plasma ceruloplasmin in burned children during total parenteral nutrition. Nutrition. 1993 Jul-Aug; 9(4):329-32. Cunningham JJ, Leffell M, Harmatz P. PMID: 8400588.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    191. Differential regulation of B7 mRNA in enterocytes and lymphoid cells. Immunology. 1993 Jul; 79(3):434-8. Sanderson IR, Ouellette AJ, Carter EA, Walker WA, Harmatz PR. PMID: 7691725; PMCID: PMC1421973.
      View in: PubMed   Mentions: 15     Fields:    Translation:AnimalsCells
    192. Intestinal adaptation during lactation in the mouse. II. Altered intestinal processing of a dietary protein. Am J Physiol. 1993 Jun; 264(6 Pt 1):G1126-32. Harmatz PR, Carrington PW, Giovino-Barry V, Hatz RA, Bloch KJ. PMID: 8333541.
      View in: PubMed   Mentions: 2     Fields:    Translation:Animals
    193. Ontogeny of class II MHC mRNA in the mouse small intestinal epithelium. Mol Immunol. 1992 Oct; 29(10):1257-63. Sanderson IR, Ouellette AJ, Carter EA, Harmatz PR. PMID: 1326714.
      View in: PubMed   Mentions: 3     Fields:    Translation:AnimalsCells
    194. Effect of thermal injury on transfer of IR22 IgA myeloma protein into bile in the rat. Liver. 1992 Jun; 12(3):124-31. Cappeller WA, Bloch KJ, Fagundes J, Carter EA, Sullivan D, Harmatz PR. PMID: 1501516.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    195. Reduction in biliary IgA after burn injury. Role of diminished delivery via the thoracic duct and of enhanced loss from the systemic circulation. Ann Surg. 1992 Apr; 215(4):338-43. Cappeller WA, Bloch KJ, Hatz RA, Carter EA, Fagundes J, Sullivan DA, Harmatz PR. PMID: 1558414; PMCID: PMC1242449.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    196. Expression of MHC class II (Ia) antigen by the neonatal enterocyte: the effect of treatment with interferon-gamma. Immunology. 1991 Apr; 72(4):491-6. Hughes A, Bloch KJ, Bhan AK, Gillen D, Giovino VC, Harmatz PR. PMID: 1903764; PMCID: PMC1384366.
      View in: PubMed   Mentions: 5     Fields:    Translation:Animals
    197. Transfer of enterally administered proteins from lactating mouse to neonate: the potential role of environmental contamination. Adv Exp Med Biol. 1991; 310:411-5. Murphy MS, Bloch KJ, Giovino V, Harmatz PR. PMID: 1725571.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    198. Major histocompatibility complex class II expression on enterocytes: to present or not to present. Gastroenterology. 1991 Jan; 100(1):274-6. Russell GJ, Harmatz PR. PMID: 1983833.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimals
    199. Divalent hapten-induced intestinal anaphylaxis in the mouse enhances macromolecular uptake from the stomach. Gastroenterology. 1990 Apr; 98(4):894-900. Hatz RA, Bloch KJ, Harmatz PR, Gonnella PA, Ariniello PD, Walker WA, Kleinman RE. PMID: 2107115.
      View in: PubMed   Mentions: 4     Fields:    Translation:AnimalsCellsPHPublic Health
    200. Divalent hapten-induced intestinal anaphylaxis in the mouse: uptake and characterization of a bystander protein. Immunology. 1989 Dec; 68(4):464-8. Kleinman RE, Harmatz PR, Hatz RA, Brown M, Ariniello PD, Walker WA, Bloch KJ. PMID: 2514138; PMCID: PMC1385531.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    201. Effect of thermal injury in the rat on transfer of IgA protein into bile. Ann Surg. 1989 Aug; 210(2):203-7. Harmatz PR, Carter EA, Sullivan D, Hatz RA, Baker R, Breazeale E, Grant K, Bloch KJ. PMID: 2757421; PMCID: PMC1357829.
      View in: PubMed   Mentions: 3     Fields:    Translation:Animals
    202. Prolactin is transported across the epithelium of the jejunum and ileum of the suckling rat. J Cell Physiol. 1989 Jul; 140(1):138-49. Gonnella PA, Harmatz P, Walker WA. PMID: 2738108.
      View in: PubMed   Mentions: 7     Fields:    Translation:AnimalsCells
    203. Intestinal adaptation during lactation in the mouse. I. Enhanced intestinal uptake of dietary protein antigen. Immunology. 1989 May; 67(1):92-5. Harmatz PR, Bloch KJ, Brown M, Walker WA, Kleinman RE. PMID: 2737698; PMCID: PMC1385294.
      View in: PubMed   Mentions: 3     Fields:    Translation:Animals
    204. Transfer of dietary protein in breast milk. Ann Allergy. 1988 Dec; 61(6 Pt 2):21-4. Harmatz PR, Bloch KJ. PMID: 3061317.
      View in: PubMed   Mentions: 1     Fields:    Translation:HumansAnimals
    205. Trichloroacetic acid (TCA) precipitability of 125I in the blood of mice fed 125I. J Immunol Methods. 1987 Sep 24; 102(2):213-9. Harmatz PR, Walsh MK, Walker WA, Hanson DG, Bloch KJ. PMID: 3655373.
      View in: PubMed   Mentions: 2     Fields:    Translation:AnimalsCells
    206. Structural and functional maturation of rat gastrointestinal barrier with thyroxine. Am J Physiol. 1987 Jun; 252(6 Pt 1):G762-7. Israel EJ, Pang KY, Harmatz PR, Walker WA. PMID: 3035939.
      View in: PubMed   Mentions: 6     Fields:    Translation:AnimalsCells
    207. Clearance, localization and catabolism of intravenously administered protein antigens in lactating mice. Adv Exp Med Biol. 1987; 216A:363-8. Harmatz PR, Hanson DG, Walsh MK, Kleinman RE, Bloch KJ, Walker WA. PMID: 2446468.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    208. Biliary IgA content following common duct cannulation in the rat. Adv Exp Med Biol. 1987; 216B:1133-8. Seo JK, Grant KE, Sullivan DA, Hanson DG, Bloch KJ, Harmatz PR, Walker WA. PMID: 3425421.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    209. Barrier defense function of the small intestine: effect of ethanol and acute burn trauma. Adv Exp Med Biol. 1987; 216A:829-33. Carter EA, Harmatz PR, Udall JN, Walker WA. PMID: 3687557.
      View in: PubMed   Mentions: 1     Fields:    Translation:Animals
    210. Transfer of protein antigens into milk after intravenous injection into lactating mice. Am J Physiol. 1986 Aug; 251(2 Pt 1):E227-33. Harmatz PR, Hanson DG, Walsh MK, Kleinman RE, Bloch KJ, Walker WA. PMID: 2426966.
      View in: PubMed   Mentions: 1     Fields:    Translation:Animals
    211. Influence of circulating maternal antibody on the transfer of dietary antigen to neonatal mice via milk. Immunology. 1986 Jan; 57(1):43-8. Harmatz PR, Bloch KJ, Kleinman RE, Walsh MK, Walker WA. PMID: 3943877; PMCID: PMC1453878.
      View in: PubMed   Mentions:    Fields:    Translation:Animals
    212. Food proteins and gut mucosal barrier. III. The influence of lactation and prolactin on the in vitro binding and uptake of bovine serum albumin and ovalbumin in the rat jejunum. Pediatr Res. 1985 Mar; 19(3):320-4. Stern M, Harmatz PR, Kleinman RE, Walker WA. PMID: 3982893.
      View in: PubMed   Mentions: 1     Fields:    Translation:Animals
    213. Sonographic demonstration of reversible portal vein thrombosis following splenectomy in an adolescent. J Clin Ultrasound. 1984 Nov-Dec; 12(9):575-7. Papanicolaou N, Harmatz P, Simeone JF, Truman JT, Ferrucci JT. PMID: 6439752.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    214. The effect of bile duct obstruction on the clearance of circulating IgA immune complexes. Hepatology. 1984 Jan-Feb; 4(1):96-100. Harmatz PR, Kleinman RE, Bunnell BW, McClenathan DT, Walker WA, Bloch KJ. PMID: 6693069.
      View in: PubMed   Mentions: 2     Fields:    Translation:Animals
    215. Passive transplacental immunization: influence on the detection of enteric antigen in the systemic circulation. Pediatr Res. 1983 Jun; 17(6):449-51. Kleinman RE, Harmatz PR, Jacobson LA, Udall JN, Bloch KJ, Walker WA. PMID: 6877896.
      View in: PubMed   Mentions: 4     Fields:    Translation:Animals
    216. The liver: an integral part of the enteric mucosal immune system. Hepatology. 1982 May-Jun; 2(3):379-84. Kleinman RE, Harmatz PR, Walker WA. PMID: 7042507.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansAnimals
    217. Hepatobiliary clearance of IgA immune complexes formed in the circulation. Hepatology. 1982 May-Jun; 2(3):328-33. Harmatz PR, Kleinman RE, Bunnell BW, Bloch KJ, Walker WA. PMID: 7076115.
      View in: PubMed   Mentions: 7     Fields:    Translation:Animals
    218. Successful treatment of primary amebic meningoencephalitis. N Engl J Med. 1982 Feb 11; 306(6):346-8. Seidel JS, Harmatz P, Visvesvara GS, Cohen A, Edwards J, Turner J. PMID: 7054710.
      View in: PubMed   Mentions: 64     Fields:    Translation:Humans
    219. The genetics of pheromonally mediated aggression in mice. I. Strain difference in the capacity of male urinary odors to elicit aggression. Behav Genet. 1975 Jul; 5(3):233-8. Kessler S, Harmatz P, Gerling SA. PMID: 1169056.
      View in: PubMed   Mentions: 4     Fields:    Translation:HumansAnimals
    220. Postisolation aggression and olfactory cues. Behav Biol. 1975 Feb; 13(2):219-24. Harmatz P, Boelkins RC, Kessler S. PMID: 1168455.
      View in: PubMed   Mentions:    Fields:    Translation:HumansAnimals
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