Paul Harmatz, MD

Title(s)Professor, Pediatrics
SchoolSchool of Medicine
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    TRANSPORT OF IGA IMMUNE COMPLEXES INTO BREAST MILK
    NIH K08AM001141Apr 1, 1983 - Mar 31, 1986
    Role: Principal Investigator

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    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. Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy. Mol Genet Metab. 2021 Mar 14. Garcia P, Phillips D, Johnson J, Martin K, Randolph LM, Rosenfeld H, Harmatz P. PMID: 33775523.
      View in: PubMed   Mentions:    Fields:    
    2. 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: 5     Fields:    Translation:HumansCTClinical Trials
    3. 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:    Fields:    Translation:Humans
    4. 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:    
    5. 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:    Fields:    
    6. 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.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    7. 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: 3     Fields:    Translation:HumansCTClinical Trials
    8. 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.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    9. 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: 2     Fields:    Translation:Humans
    10. 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.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCTClinical Trials
    11. 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.
      View in: PubMed   Mentions:
    12. 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: 23     Fields:    Translation:HumansCTClinical Trials
    13. 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.
      View in: PubMed   Mentions: 17     Fields:    Translation:Humans
    14. 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.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    15. 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.
      View in: PubMed   Mentions: 2     Fields:    Translation:Humans
    16. 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: 2     Fields:    Translation:Humans
    17. 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: 1     Fields:    Translation:Humans
    18. 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.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCTClinical Trials
    19. 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: 9     Fields:    Translation:Humans
    20. 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: 2     Fields:    Translation:HumansCTClinical Trials
    21. 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.
      View in: PubMed   Mentions:
    22. 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: 18     Fields:    Translation:HumansCTClinical Trials
    23. 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.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    24. 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: 8     Fields:    Translation:HumansCTClinical Trials
    25. 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.
      View in: PubMed   Mentions:
    26. 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: 3     Fields:    Translation:HumansCells
    27. 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: 23     Fields:    Translation:Humans
    28. 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: 12     Fields:    Translation:Humans
    29. 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.
      View in: PubMed   Mentions: 18     Fields:    Translation:Humans
    30. Mucopolysaccharidosis VI: pathophysiology, diagnosis and treatment. Front Biosci (Landmark Ed). 2017 01 01; 22:385-406. Harmatz P, Shediac R. PMID: 27814620.
      View in: PubMed   Mentions: 20     Fields:    Translation:Humans
    31. 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: 9     Fields:    Translation:HumansCTClinical Trials
    32. 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.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCTClinical Trials
    33. 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.
      View in: PubMed   Mentions:
    34. 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.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansCTClinical Trials
    35. 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: 21     Fields:    Translation:HumansCTClinical Trials
    36. 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: 5     Fields:    Translation:HumansCTClinical Trials
    37. 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: 15     Fields:    Translation:Humans
    38. 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
    39. 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.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCTClinical Trials
    40. 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: 10     Fields:    Translation:Humans
    41. 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.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCTClinical Trials
    42. 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.
      View in: PubMed   Mentions: 25     Fields:    Translation:HumansCTClinical Trials
    43. 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.
      View in: PubMed   Mentions: 14     Fields:    Translation:Humans
    44. 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: 2     Fields:    Translation:HumansCTClinical Trials
    45. 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.
      View in: PubMed   Mentions:
    46. 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.
      View in: PubMed   Mentions: 14     Fields:    Translation:HumansCTClinical Trials
    47. 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.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
    48. 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: 14     Fields:    Translation:Humans
    49. International guidelines for the management and treatment of Morquio A syndrome. . 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.
      View in: PubMed   Mentions:
    50. 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.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansAnimalsPHPublic Health
    51. 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.
      View in: PubMed   Mentions: 13     Fields:    Translation:HumansCTClinical Trials
    52. 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: 31     Fields:    Translation:HumansCTClinical Trials
    53. 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.
      View in: PubMed   Mentions:
    54. 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:    Fields:    Translation:HumansAnimals
    55. 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.
      View in: PubMed   Mentions: 73     Fields:    Translation:HumansCTClinical Trials
    56. 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. . 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.
      View in: PubMed   Mentions:
    57. 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.
      View in: PubMed   Mentions: 8     Fields:    Translation:HumansCTClinical Trials
    58. 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.
      View in: PubMed   Mentions: 15     Fields:    Translation:Humans
    59. 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.
      View in: PubMed   Mentions: 11     Fields:    Translation:Humans
    60. 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.
      View in: PubMed   Mentions: 36     Fields:    Translation:Humans
    61. 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.
      View in: PubMed   Mentions:
    62. 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: 27     Fields:    Translation:Humans
    63. 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: 7     Fields:    Translation:Humans
    64. 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: 46     Fields:    Translation:Humans
    65. 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.
      View in: PubMed   Mentions: 29     Fields:    Translation:Humans
    66. 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.
      View in: PubMed   Mentions: 36     Fields:    Translation:HumansCells
    67. 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.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCTClinical Trials
    68. 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: 4     Fields:    Translation:Humans
    69. Increased leucocyte apoptosis in transfused ß-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.
      View in: PubMed   Mentions: 2     Fields:    Translation:HumansCells
    70. 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.
      View in: PubMed   Mentions: 34     Fields:    Translation:Humans
    71. 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.
      View in: PubMed   Mentions: 15     Fields:    Translation:HumansCTClinical Trials
    72. 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: 10     Fields:    Translation:Humans
    73. 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.
      View in: PubMed   Mentions: 10     Fields:    Translation:HumansCTClinical Trials
    74. 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: 21     Fields:    Translation:Humans
    75. 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.
      View in: PubMed   Mentions: 13     Fields:    Translation:Humans
    76. 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.
      View in: PubMed   Mentions: 32     Fields:    Translation:Humans
    77. 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: 18     Fields:    Translation:Humans
    78. 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.
      View in: PubMed   Mentions: 1     Fields:    Translation:Humans
    79. 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.
      View in: PubMed   Mentions: 84     Fields:    Translation:Humans
    80. 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.
      View in: PubMed   Mentions: 12     Fields:    Translation:HumansCTClinical Trials
    81. 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: 72     Fields:    Translation:Humans
    82. 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.
      View in: PubMed   Mentions: 11     Fields:    Translation:HumansCTClinical Trials
    83. 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: 30     Fields:    Translation:Humans
    84. 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
    85. 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: 15     Fields:    Translation:Humans
    86. 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: 4     Fields:    Translation:Humans
    87. 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: 21     Fields:    Translation:Humans
    88. 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: 37     Fields:    Translation:HumansCTClinical Trials
    89. Mucopolysaccharidosis VI. Orphanet J Rare Dis. 2010 Apr 12; 5:5. Valayannopoulos V, Nicely H, Harmatz P, Turbeville S. PMID: 20385007.
      View in: PubMed   Mentions: 82     Fields:    Translation:Humans
    90. 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: 5     Fields:    Translation:Humans
    91. 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.
      View in: PubMed   Mentions: 24     Fields:    Translation:Humans
    92. 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.
      View in: PubMed   Mentions: 8     Fields:    Translation:Humans
    93. 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.
      View in: PubMed   Mentions: 19     Fields:    
    94. Orthopedic management of mucopolysaccharide disease. J Pediatr Rehabil Med. 2010; 3(1):47-56. White KK, Harmatz P. PMID: 21791829.
      View in: PubMed   Mentions: 17     Fields:    
    95. 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.
      View in: PubMed   Mentions: 26     Fields:    
    96. 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: 18     Fields:    Translation:Humans
    97. 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.
      View in: PubMed   Mentions: 9     Fields:    Translation:HumansCells
    98. 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: 53     Fields:    Translation:HumansCTClinical Trials
    99. 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
    100. 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: 27     Fields:    Translation:Humans
    101. 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.
      View in: PubMed   Mentions: 5     Fields:    Translation:Humans
    102. 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.
      View in: PubMed   Mentions: 3     Fields:    Translation:Humans
    103. 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.
      View in: PubMed   Mentions: 29     Fields:    Translation:HumansCTClinical Trials
    104. 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
    105. 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.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    106. 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):469-75. Harmatz P, 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, Yu ZF, Swiedler SJ, Decker C, MPS VI Study Group . PMID: 18502162.
      View in: PubMed   Mentions: 78     Fields:    Translation:HumansCTClinical Trials
    107. 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: 22     Fields:    Translation:HumansCellsCTClinical Trials
    108. 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.
      View in: PubMed   Mentions: 6     Fields:    Translation:Humans
    109. 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.
      View in: PubMed   Mentions: 36     Fields:    Translation:Humans
    110. 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.
      View in: PubMed   Mentions: 22     Fields:    Translation:Humans
    111. 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
    112. 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.
      View in: PubMed   Mentions: 7     Fields:    Translation:HumansCells
    113. 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: 92     Fields:    Translation:Humans
    114. 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: 7     Fields:    Translation:Humans
    115. 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: 14     Fields:    Translation:HumansCTClinical Trials
    116. 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: 30     Fields:    Translation:Humans
    117. 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: 42     Fields:    Translation:HumansCells
    118. 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: 9     Fields:    Translation:HumansCTClinical Trials
    119. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007 Aug; 120(2):405-18. Giugliani R, Harmatz P, Wraith JE. PMID: 17671068.
      View in: PubMed   Mentions: 57     Fields:    Translation:Humans
    120. 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: 12     Fields:    Translation:Humans
    121. 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: 43     Fields:    Translation:Humans
    122. 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: 10     Fields:    Translation:HumansCells
    123. 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: 14     Fields:    Translation:HumansCTClinical Trials
    124. 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: 7     Fields:    Translation:HumansCTClinical Trials
    125. 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: 39     Fields:    Translation:Humans
    126. 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.
      View in: PubMed   Mentions: 72     Fields:    Translation:Humans
    127. 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
    128. 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: 188     Fields:    Translation:HumansCTClinical Trials
    129. 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
    130. 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: 111     Fields:    Translation:HumansCTClinical Trials
    131. 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: 36     Fields:    Translation:Humans
    132. 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.
      View in: PubMed   Mentions: 10     Fields:    Translation:Humans
    133. 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: 63     Fields:    Translation:HumansCTClinical Trials
    134. 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). . 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:
    135. 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
    136. 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: 4     Fields:    Translation:Humans
    137. 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
    138. 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: 20     Fields:    Translation:Humans
    139. 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:    Fields:    Translation:Humans
    140. 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: 11     Fields:    Translation:Humans
    141. 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: 80     Fields:    Translation:HumansCTClinical Trials
    142. Bioelectrical impedance analysis of the body composition of children and adolescents with sickle cell disease. J Pediatr. 2002 Jun; 140(6):681-7. VanderJagt DJ, Harmatz P, Scott-Emuakpor AB, Vichinsky E, Glew RH. PMID: 12072870.
      View in: PubMed   Mentions: 4     Fields:    Translation:Humans
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