Paul Harmatz, MD

  1. Harmatz P, Muenzer J, Ezgü F, Dalén P, Huledal G, Lindqvist D, Gelius SS, Wikén M, Önnestam K, Bröijersén A. Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study. Molecular genetics and metabolism 2022. PMID: 35835061


  2. 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. Longitudinal natural history of pediatric subjects affected with mucopolysaccharidosis IIIB. The Journal of pediatrics 2022. PMID: 35709957


  3. Sohn YB, Rogers C, Stallworth J, Cooley Coleman JA, Buch L, Jozwiak E, Johnson JA, Wood T, Harmatz P, Pollard L, Louie RJ. RNA analysis of the GALNS transcript reveals novel pathogenic mechanisms associated with Morquio syndrome A. Molecular genetics and metabolism reports 2022. PMID: 35782621


  4. 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. Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. 2022. PMID: 35342457


  5. Grant N, Sohn YB, Ellinwood NM, Okenfuss E, Mendelsohn BA, Lynch LE, Braunlin EA, Harmatz PR, Eisengart JB. Timing is everything: Clinical courses of Hunter syndrome associated with age at initiation of therapy in a sibling pair. 2022. PMID: 35242576


  6. Schwab ME, Brown JEH, Lianoglou B, Jin C, Conroy PC, Gallagher RC, Harmatz P, MacKenzie TC. Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients. 2022. PMID: 35093147


  7. 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. Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I. 2022. PMID: 35012890




  8. 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. 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. Journal of inherited metabolic disease 2021. PMID: 34418116


  9. 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. Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study. Genetics in medicine : official journal of the American College of Medical Genetics 2021. PMID: 34341520



  10. 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. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet (London, England) 2020. PMID: 32891212


  11. 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. Therapy development for the mucopolysaccharidoses: Updated consensus recommendations for neuropsychological endpoints. Molecular genetics and metabolism 2020. PMID: 32917509


  12. 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. 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]. Molecular genetics and metabolism 2020. PMID: 32843286


  13. 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. ASAH1 pathogenic variants associated with acid ceramidase deficiency: Farber disease and spinal muscular atrophy with progressive myoclonic epilepsy. Human mutation 2020. PMID: 32449975



  14. 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. The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII. Molecular genetics and metabolism 2020. PMID: 32063397


  15. 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. Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study. Molecular genetics and metabolism 2019. PMID: 31839529



  16. 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. Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study. The Journal of clinical endocrinology and metabolism 2019. PMID: 30811537



  17. 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. C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia. The New England journal of medicine 2019. PMID: 31269546


  18. 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. Recommendations for the management of MPS IVA: systematic evidence- and consensus-based guidance. Orphanet journal of rare diseases 2019. PMID: 31196221


  19. 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. Recommendations for the management of MPS VI: systematic evidence- and consensus-based guidance. Orphanet journal of rare diseases 2019. PMID: 31142378


  20. 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. Attention and corpus callosum volumes in individuals with mucopolysaccharidosis type I. Neurology 2019. PMID: 30979856




  21. 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. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. Journal of inherited metabolic disease 2018. PMID: 29725868



  22. Whitley CB, Cleary M, Eugen Mengel K, Harmatz P, Shapiro E, Nestrasil I, Haslett P, Whiteman D, Alexanderian D. Observational Prospective Natural History of Patients with Sanfilippo Syndrome Type B. The Journal of pediatrics 2018. PMID: 29661560





  23. 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. 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. Molecular genetics and metabolism 2017. PMID: 29248359


  24. 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. 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 reports 2017. PMID: 29159458


  25. Alden TD, Amartino H, Dalla Corte A, Lampe C, Harmatz PR, Vedolin L. Surgical management of neurological manifestations of mucopolysaccharidosis disorders. Molecular genetics and metabolism 2017. PMID: 29153846


  26. 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. Monitoring guidance for patients with hypophosphatasia treated with asfotase alfa. Molecular genetics and metabolism 2017. PMID: 28888853


  27. 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. Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure. Molecular genetics and metabolism 2017. PMID: 28501294


  28. 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. Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry. Orphanet journal of rare diseases 2017. PMID: 28464912


  29. 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. The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Molecular genetics and metabolism 2017. PMID: 28457718


  30. Giugliani R, Harmatz P, Jones SA, Mendelsohn NJ, Vellodi A, Qiu Y, Hendriksz CJ, Vijayaraghavan S, Whiteman DA, Pano A. Evaluation of impact of anti-idursulfase antibodies during long-term idursulfase enzyme replacement therapy in mucopolysaccharidosis II patients. Molecular genetics and metabolism reports 2017. PMID: 28243577



  31. Long B, Tompkins T, Decker C, Jesaitis L, Khan S, Slasor P, Harmatz P, O'Neill CA, Schweighardt B. Long-term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study. Clinical therapeutics 2016. PMID: 27955919


  32. Harmatz PR, Mengel E, Geberhiwot T, Muschol N, Hendriksz CJ, Burton BK, Jameson E, Berger KI, Jester A, Treadwell M, Sisic Z, Decker C. Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open-label, phase 2 study. American journal of medical genetics. Part A 2016. PMID: 27774754


  33. 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. Pregnancy in patients with mucopolysaccharidosis: a case series. Molecular genetics and metabolism reports 2016. PMID: 27622143


  34. 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. Impact of long-term elosulfase alfa treatment on respiratory function in patients with Morquio A syndrome. Journal of inherited metabolic disease 2016. PMID: 27553181


  35. 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. Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome. Molecular genetics and metabolism 2016. PMID: 27380995


  36. Solanki GA, Sun PP, Martin KW, Hendriksz CJ, Lampe C, Guffon N, Hung A, Sisic Z, Shediac R, Harmatz PR, CSP Study Group. Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP). Molecular genetics and metabolism 2016. PMID: 27339555




  37. Jones SA, Bialer M, Parini R, Martin K, Wang H, Yang K, Shaywitz AJ, Harmatz P. Safety and clinical activity of elosulfase alfa in pediatric patients with Morquio A syndrome (mucopolysaccharidosis IVA) less than 5 y. Pediatric research 2015. PMID: 26331768


  38. 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. Data from subjects receiving intrathecal laronidase for cervical spinal stenosis due to mucopolysaccharidosis type I. Data in brief 2015. PMID: 26484358



  39. 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. Safety of laronidase delivered into the spinal canal for treatment of cervical stenosis in mucopolysaccharidosis I. Molecular genetics and metabolism 2015. PMID: 26260077


  40. 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. Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment. Molecular genetics and metabolism 2015. PMID: 26095521


  41. 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. Safety and physiological effects of two different doses of elosulfase alfa in patients with morquio a syndrome: A randomized, double-blind, pilot study. American journal of medical genetics. Part A 2015. PMID: 26069231


  42. Smith L, Rhead W, Charrow J, Shankar SP, Bavdekar A, Longo N, Mardach R, Harmatz P, Hangartner T, Lee HM, Crombez E, Pastores GM. Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase. Molecular genetics and metabolism 2015. PMID: 26043810


  43. Quartel A, Hendriksz CJ, Parini R, Graham S, Lin P, Harmatz P. Growth Charts for Individuals with Mucopolysaccharidosis VI (Maroteaux-Lamy Syndrome). JIMD reports 2014. PMID: 25518809


  44. 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. Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type II. Molecular genetics and metabolism 2014. PMID: 25541100


  45. Meloni A, Detterich J, Pepe A, Harmatz P, Coates TD, Wood JC. Pulmonary hypertension in well-transfused thalassemia major patients. Blood cells, molecules & diseases 2014. PMID: 25488617


  46. 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. Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome. Molecular genetics and metabolism 2014. PMID: 25582974


  47. Hendriksz CJ, Berger KI, Giugliani R, Harmatz P, Kampmann C, Mackenzie WG, Raiman J, Villarreal MS, Savarirayan R. International guidelines for the management and treatment of Morquio A syndrome. American journal of medical genetics. Part A 2014. PMID: 25346323


  48. 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. Morquio A syndrome-associated mutations: a review of alterations in the GALNS gene and a new locus-specific database. Human mutation 2014. PMID: 25137622


  49. 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. Mechanisms of plasma non-transferrin bound iron generation: insights from comparing transfused diamond blackfan anaemia with sickle cell and thalassaemia patients. British journal of haematology 2014. PMID: 25209728


  50. 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. Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial. Molecular genetics and metabolism 2014. PMID: 25284089


  51. White KK, Jester A, Bache CE, Harmatz PR, Shediac R, Thacker MM, Mackenzie WG. Orthopedic management of the extremities in patients with Morquio A syndrome. Journal of children's orthopaedics 2014. PMID: 25001525



  52. 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. 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. Journal of inherited metabolic disease 2014. PMID: 24810369



  53. Harmatz PR, Garcia P, Guffon N, Randolph LM, Shediac R, Braunlin E, Lachman RS, Decker C. Galsulfase (Naglazyme®) therapy in infants with mucopolysaccharidosis VI. Journal of inherited metabolic disease 2013. PMID: 24108527


  54. 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. Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure. Orphanet journal of rare diseases 2013. PMID: 24088413


  55. Vera M, Le S, Kan SH, Garban H, Naylor D, Mlikotic A, Kaitila I, Harmatz P, Chen A, Dickson P. Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients. Pediatric research 2013. PMID: 24002329


  56. 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. 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. PMID: 23825616



  57. Hendriksz CJ, Harmatz P, Beck M, Jones S, Wood T, Lachman R, Gravance CG, Orii T, Tomatsu S. Review of clinical presentation and diagnosis of mucopolysaccharidosis IVA. Molecular genetics and metabolism 2013. PMID: 23665161


  58. Jones SA, Parini R, Harmatz P, Giugliani R, Fang J, Mendelsohn NJ, HOS Natural History Working Group on behalf of HOS Investigators. The effect of idursulfase on growth in patients with Hunter syndrome: data from the Hunter Outcome Survey (HOS). Molecular genetics and metabolism 2013. PMID: 23537841



  59. 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. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. Molecular genetics and metabolism 2013. PMID: 23452954


  60. Solanki GA, Martin KW, Theroux MC, Lampe C, White KK, Shediac R, Lampe CG, Beck M, Mackenzie WG, Hendriksz CJ, Harmatz PR. Spinal involvement in mucopolysaccharidosis IVA (Morquio-Brailsford or Morquio A syndrome): presentation, diagnosis and management. Journal of inherited metabolic disease 2013. PMID: 23385297


  61. 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. Diagnosing mucopolysaccharidosis IVA. Journal of inherited metabolic disease 2013. PMID: 23371450


  62. Sohn EY, Kato R, Noetzli LJ, Gera A, Coates T, Harmatz P, Keens TG, Wood JC. Exercise performance in thalassemia major: correlation with cardiac iron burden. American journal of hematology 2013. PMID: 23339082


  63. 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. Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase. American journal of hematology 2013. PMID: 23339116


  64. 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. Increased leucocyte apoptosis in transfused ß-thalassaemia patients. British journal of haematology 2012. PMID: 23216540


  65. Walker R, Belani KG, Braunlin EA, Bruce IA, Hack H, Harmatz PR, Jones S, Rowe R, Solanki GA, Valdemarsson B. Anaesthesia and airway management in mucopolysaccharidosis. Journal of inherited metabolic disease 2012. PMID: 23197104


  66. Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E. Combined chelation therapy with deferasirox and deferoxamine in thalassemia. Blood cells, molecules & diseases 2012. PMID: 23151373


  67. 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. A multinational, multidisciplinary consensus for the diagnosis and management of spinal cord compression among patients with mucopolysaccharidosis VI. Molecular genetics and metabolism 2012. PMID: 22938833


  68. 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. Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme®) therapy. Journal of inherited metabolic disease 2012. PMID: 22669363


  69. 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. Expert recommendations for the laboratory diagnosis of MPS VI. Molecular genetics and metabolism 2012. PMID: 22405600


  70. 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. A phase 2 study of the safety, tolerability, and pharmacodynamics of FBS0701, a novel oral iron chelator, in transfusional iron overload. Blood 2012. PMID: 22251482


  71. Hendriksz CJ, Giugliani R, Harmatz P, Lampe C, Martins AM, Pastores GM, Steiner RD, Leão Teles E, Valayannopoulos V, CSP Study Group. Design, baseline characteristics, and early findings of the MPS VI (mucopolysaccharidosis VI) Clinical Surveillance Program (CSP). Journal of inherited metabolic disease 2011. PMID: 22127392


  72. Detterich J, Noetzli L, Dorey F, Bar-Cohen Y, Harmatz P, Coates T, Wood J. Electrocardiographic consequences of cardiac iron overload in thalassemia major. American journal of hematology 2011. PMID: 22052662


  73. Muenzer J, Bodamer O, Burton B, Clarke L, Frenking GS, Giugliani R, Jones S, Rojas MV, Scarpa M, Beck M, Harmatz P. The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus. European journal of pediatrics 2011. PMID: 22037758


  74. 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. Iron chelation therapy in thalassemia major: a systematic review with meta-analyses of 1520 patients included on randomized clinical trials. Blood cells, molecules & diseases 2011. PMID: 21843958


  75. Sohn EY, Noetzli LJ, Gera A, Kato R, Coates TD, Harmatz P, Keens TG, Wood JC. Pulmonary function in thalassaemia major and its correlation with body iron stores. British journal of haematology 2011. PMID: 21810090


  76. Braunlin EA, Harmatz PR, Scarpa M, Furlanetto B, Kampmann C, Loehr JP, Ponder KP, Roberts WC, Rosenfeld HM, Giugliani R. Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management. Journal of inherited metabolic disease 2011. PMID: 21744090


  77. Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial. Haematologica 2011. PMID: 21393329


  78. 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. Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome. Genetics in medicine : official journal of the American College of Medical Genetics 2011. PMID: 21150784


  79. Rienhoff HY, Viprakasit V, Tay L, Harmatz P, Vichinsky E, Chirnomas D, Kwiatkowski JL, Tapper A, Kramer W, Porter JB, Neufeld EJ. 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 2010. PMID: 21173101


  80. Mendelsohn NJ, Harmatz P, Bodamer O, Burton BK, Giugliani R, Jones SA, Lampe C, Malm G, Steiner RD, Parini R, Hunter Outcome Survey Investigators. Importance of surgical history in diagnosing mucopolysaccharidosis type II (Hunter syndrome): data from the Hunter Outcome Survey. Genetics in medicine : official journal of the American College of Medical Genetics 2010. PMID: 21045710


  81. Wood JC, Glynos T, Thompson A, Giardina P, Harmatz P, Kang BP, Paley C, Coates TD. Follow-up report on the 2-year cardiac data from a deferasirox monotherapy trial. American journal of hematology 2010. PMID: 20799360


  82. Ferrari S, Ponzin D, Ashworth JL, Fahnehjelm KT, Summers CG, Harmatz PR, Scarpa M. Diagnosis and management of ophthalmological features in patients with mucopolysaccharidosis. The British journal of ophthalmology 2010. PMID: 20852315



  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. Management of chronic viral hepatitis in patients with thalassemia: recommendations from an international panel. Blood 2010. PMID: 20551378


  84. Wood JC, Kang BP, Thompson A, Giardina P, Harmatz P, Glynos T, Paley C, Coates TD. The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores. Blood 2010. PMID: 20421452


  85. Valayannopoulos V, Nicely H, Harmatz P, Turbeville S. Mucopolysaccharidosis VI. Orphanet journal of rare diseases 2010. PMID: 20385007


  86. James EB, Vreman HJ, Wong RJ, Stevenson DK, Vichinsky E, Schumacher L, Hall JY, Simon J, Golden DW, Harmatz P. Elevated exhaled carbon monoxide concentration in hemoglobinopathies and its relation to red blood cell transfusion therapy. Pediatric hematology and oncology 2010. PMID: 20201692


  87. 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. Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. Journal of inherited metabolic disease 2010. PMID: 20140523


  88. Wang ZJ, Fischer R, Chu Z, Mahoney DH, Mueller BU, Muthupillai R, James EB, Krishnamurthy R, Chung T, Padua E, Vichinsky E, Harmatz P. Assessment of cardiac iron by MRI susceptometry and R2* in patients with thalassemia. Magnetic resonance imaging 2010. PMID: 20061110



  89. 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. Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase. Journal of pediatric rehabilitation medicine 2010. PMID: 20634905



  90. Walter PB, Harmatz P, Vichinsky E. Iron metabolism and iron chelation in sickle cell disease. Acta haematologica 2009. PMID: 19907155


  91. 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. Multidisciplinary management of Hunter syndrome. Pediatrics 2009. PMID: 19901005


  92. Ghugre NR, Gonzalez-Gomez I, Butensky E, Noetzli L, Fischer R, Williams R, Harmatz P, Coates TD, Wood JC. Patterns of hepatic iron distribution in patients with chronically transfused thalassemia and sickle cell disease. American journal of hematology 2009. PMID: 19536851


  93. 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. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. The Journal of pediatrics 2009. PMID: 19261295


  94. Butensky James E, Harmatz P, Lee M, Kennedy C, Petru A, Wara D, Miaskowski C. Altered iron metabolism in children with human immunodeficiency virus disease. Pediatric hematology and oncology 2009. PMID: 19322737



  95. 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. Folate concentrations in pediatric patients with newly diagnosed inflammatory bowel disease. The American journal of clinical nutrition 2008. PMID: 19116333


  96. Nielsen P, Engelhardt R, Grosse R, Janka G, Harmatz P, Fischer R. Italian Society of Hematology guidelines for thalassemia and non-invasive iron measurements. Haematologica 2008. PMID: 19109216


  97. Block G, Jensen CD, Dalvi TB, Norkus EP, Hudes M, Crawford PB, Holland N, Fung EB, Schumacher L, Harmatz P. Vitamin C treatment reduces elevated C-reactive protein. Free radical biology & medicine 2008. PMID: 18952164


  98. Harmatz P, Jonas MM, Kwiatkowski JL, Wright EC, Fischer R, Vichinsky E, Giardina PJ, Neufeld EJ, Porter J, Olivieri N, Thalassemia Clinical Research Network. Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia. Haematologica 2008. PMID: 18556414


  99. 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. Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions. Transfusion 2008. PMID: 18513257


  100. 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. 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. Molecular genetics and metabolism 2008. PMID: 18502162


  101. 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. 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. PMID: 18469351


  102. Koseoglu ST, Harmatz P, Turbeville S, Nicely H. Reversed papilledema in an MPS VI patient with galsulfase (Naglazyme) therapy. International ophthalmology 2008. PMID: 18418554


  103. Block G, Jensen CD, Morrow JD, Holland N, Norkus EP, Milne GL, Hudes M, Dalvi TB, Crawford PB, Fung EB, Schumacher L, Harmatz P. The effect of vitamins C and E on biomarkers of oxidative stress depends on baseline level. Free radical biology & medicine 2008. PMID: 18455517


  104. 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. Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia. Bone 2008. PMID: 18430624



  105. 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. 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. Pediatric research 2008. PMID: 18287963


  106. Martin R, Beck M, Eng C, Giugliani R, Harmatz P, Muñoz V, Muenzer J. Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics 2008. PMID: 18245410



  107. 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. Pharmacokinetics of sapropterin in patients with phenylketonuria. Clinical pharmacokinetics 2008. PMID: 19026037


  108. 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. Mutational analysis of 105 mucopolysaccharidosis type VI patients. Human mutation 2007. PMID: 17458871



  109. Giugliani R, Harmatz P, Wraith JE. Management guidelines for mucopolysaccharidosis VI. Pediatrics 2007. PMID: 17671068


  110. Harmatz P, Grady RW, Dragsten P, Vichinsky E, Giardina P, Madden J, Jeng M, Miller B, Hanson G, Hedlund B. Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator. British journal of haematology 2007. PMID: 17614825



  111. 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. Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload. American journal of hematology 2007. PMID: 17094096


  112. Holland N, Harmatz P, Golden D, Hubbard A, Wu YY, Bae J, Chen C, Huen K, Heyman MB. Cytogenetic damage in blood lymphocytes and exfoliated epithelial cells of children with inflammatory bowel disease. Pediatric research 2007. PMID: 17237724


  113. Moskowitz JT, Butensky E, Harmatz P, Vichinsky E, Heyman MB, Acree M, Wrubel J, Wilson L, Folkman S. Caregiving time in sickle cell disease: psychological effects in maternal caregivers. Pediatric blood & cancer 2007. PMID: 16622841


  114. Karageorgos L, Brooks DA, Harmatz P, Ketteridge D, Pollard A, Melville EL, Parkinson-Lawrence E, Clements PR, Hopwood JJ. Mutational analysis of mucopolysaccharidosis type VI patients undergoing a phase II trial of enzyme replacement therapy. Molecular genetics and metabolism 2006. PMID: 17161971


  115. 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. Increased prevalence of iron-overload associated endocrinopathy in thalassaemia versus sickle-cell disease. British journal of haematology 2006. PMID: 17054676


  116. Walter PB, Fung EB, Killilea DW, Jiang Q, Hudes M, Madden J, Porter J, Evans P, Vichinsky E, Harmatz P. Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease. British journal of haematology 2006. PMID: 17010049


  117. Fischer R, Harmatz P, Nielsen P. Does liver biopsy overestimate liver iron concentration? Blood 2006. PMID: 16926297


  118. 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. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genetics in medicine : official journal of the American College of Medical Genetics 2006. PMID: 16912578


  119. Fischer R, Harmatz P, Nielsen P. Does liver biopsy overestimate liver iron concentration? Blood 2006. PMID: 16822911


  120. 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. 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-l The Journal of pediatrics 2006. PMID: 16647419


  121. Vichinsky E, Butensky E, Fung E, Hudes M, Theil E, Ferrell L, Williams R, Louie L, Lee PD, Harmatz P. Comparison of organ dysfunction in transfused patients with SCD or beta thalassemia. American journal of hematology 2005. PMID: 16138345


  122. Wilson LS, Moskowitz JT, Acree M, Heyman MB, Harmatz P, Ferrando SJ, Folkman S. The economic burden of home care for children with HIV and other chronic illnesses. American journal of public health 2005. PMID: 15985648



  123. 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. 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). American journal of medical genetics. Part A 2005. PMID: 15690405



  124. Pakbaz Z, Fischer R, Treadwell M, Yamashita R, Fung EB, Calvelli L, Quirolo K, Foote D, Harmatz P, Vichinsky EP. A simple model to assess and improve adherence to iron chelation therapy with deferoxamine in patients with thalassemia. Annals of the New York Academy of Sciences 2005. PMID: 16339703



  125. Butensky E, Pakbaz Z, Foote D, Walters MC, Vichinsky EP, Harmatz P. Treatment of hepatitis C virus infection in thalassemia. Annals of the New York Academy of Sciences 2005. PMID: 16339677


  126. Butensky E, Fischer R, Hudes M, Schumacher L, Williams R, Moyer TP, Vichinsky E, Harmatz P. Variability in hepatic iron concentration in percutaneous needle biopsy specimens from patients with transfusional hemosiderosis. American journal of clinical pathology 2005. PMID: 15762291


  127. Engelhardt R, Fung EB, Kelly P, Biehl TR, Pakbaz Z, Nielsen P, Harmatz P, Fischer R. Interaction of artificial metallic objects with biosusceptometric measurements. Neurology & clinical neurophysiology : NCN 2004. PMID: 16012652


  128. Fung EB, Fischer R, Pakbaz Z, Fagaly RL, Vichinsky E, Starr TN, Ewing T, Paulson DN, Hassenzahl WV, Harmatz P. The new SQUID biosusceptometer at Oakland: first year of experience. Neurology & clinical neurophysiology : NCN 2004. PMID: 16012600



  129. Heyman MB, Harmatz P, Acree M, Wilson L, Moskowitz JT, Ferrando S, Folkman S. Economic and psychologic costs for maternal caregivers of gastrostomy-dependent children. The Journal of pediatrics 2004. PMID: 15480377


  130. Harmatz P, Whitley CB, Waber L, Pais R, Steiner R, Plecko B, Kaplan P, Simon J, Butensky E, Hopwood JJ. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). The Journal of pediatrics 2004. PMID: 15126989


  131. Karageorgos L, Harmatz P, Simon J, Pollard A, Clements PR, Brooks DA, Hopwood JJ. Mutational analysis of mucopolysaccharidosis type VI patients undergoing a trial of enzyme replacement therapy. Human mutation 2004. PMID: 14974081


  132. VanderJagt DJ, Harmatz P, Scott-Emuakpor AB, Vichinsky E, Glew RH. Bioelectrical impedance analysis of the body composition of children and adolescents with sickle cell disease. The Journal of pediatrics 2002. PMID: 12072870




  133. Harmatz P, Butensky E, Quirolo K, Williams R, Ferrell L, Moyer T, Golden D, Neumayr L, Vichinsky E. Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy. Blood 2000. PMID: 10891433


  134. Vreman HJ, Wong RJ, Harmatz P, Fanaroff AA, Berman B, Stevenson DK. Validation of the Natus CO-Stat End Tidal Breath Analyzer in children and adults. Journal of clinical monitoring and computing 1999. PMID: 12578038


  135. Harmatz P, Heyman MB, Cunningham J, Lee PD, Styles L, Quirolo K, Kopp-Hoolihan L, Ghiron J, Hintz RL, Vichinsky E. Effects of red blood cell transfusion on resting energy expenditure in adolescents with sickle cell anemia. Journal of pediatric gastroenterology and nutrition 1999. PMID: 10435647


  136. Capano G, Bloch KJ, Carter EA, Dascoli JA, Schoenfeld D, Harmatz PR. Polyamines in human and rat milk influence intestinal cell growth in vitro. Journal of pediatric gastroenterology and nutrition 1998. PMID: 9740197







  137. Sanderson IR, Ouellette AJ, Carter EA, Walker WA, Harmatz PR. Differential regulation of B7 mRNA in enterocytes and lymphoid cells. Immunology 1993. PMID: 7691725



  138. Sanderson IR, Ouellette AJ, Carter EA, Harmatz PR. Ontogeny of class II MHC mRNA in the mouse small intestinal epithelium. Molecular immunology 1992. PMID: 1326714


  139. Cappeller WA, Bloch KJ, Fagundes J, Carter EA, Sullivan D, Harmatz PR. Effect of thermal injury on transfer of IR22 IgA myeloma protein into bile in the rat. Liver 1992. PMID: 1501516



  140. Hughes A, Bloch KJ, Bhan AK, Gillen D, Giovino VC, Harmatz PR. Expression of MHC class II (Ia) antigen by the neonatal enterocyte: the effect of treatment with interferon-gamma. Immunology 1991. PMID: 1903764




  141. Hatz RA, Bloch KJ, Harmatz PR, Gonnella PA, Ariniello PD, Walker WA, Kleinman RE. Divalent hapten-induced intestinal anaphylaxis in the mouse enhances macromolecular uptake from the stomach. Gastroenterology 1990. PMID: 2107115


  142. Kleinman RE, Harmatz PR, Hatz RA, Brown M, Ariniello PD, Walker WA, Bloch KJ. Divalent hapten-induced intestinal anaphylaxis in the mouse: uptake and characterization of a bystander protein. Immunology 1989. PMID: 2514138


  143. Harmatz PR, Carter EA, Sullivan D, Hatz RA, Baker R, Breazeale E, Grant K, Bloch KJ. Effect of thermal injury in the rat on transfer of IgA protein into bile. Annals of surgery 1989. PMID: 2757421




  144. Harmatz PR, Bloch KJ. Transfer of dietary protein in breast milk. Annals of allergy 1988. PMID: 3061317


  145. Harmatz PR, Walsh MK, Walker WA, Hanson DG, Bloch KJ. Trichloroacetic acid (TCA) precipitability of 125I in the blood of mice fed 125I. Journal of immunological methods 1987. PMID: 3655373




  146. Seo JK, Grant KE, Sullivan DA, Hanson DG, Bloch KJ, Harmatz PR, Walker WA. Biliary IgA content following common duct cannulation in the rat. Advances in experimental medicine and biology 1987. PMID: 3425421



  147. Harmatz PR, Hanson DG, Walsh MK, Kleinman RE, Bloch KJ, Walker WA. Transfer of protein antigens into milk after intravenous injection into lactating mice. The American journal of physiology 1986. PMID: 2426966


  148. Harmatz PR, Bloch KJ, Kleinman RE, Walsh MK, Walker WA. Influence of circulating maternal antibody on the transfer of dietary antigen to neonatal mice via milk. Immunology 1986. PMID: 3943877




  149. Harmatz PR, Kleinman RE, Bunnell BW, McClenathan DT, Walker WA, Bloch KJ. The effect of bile duct obstruction on the clearance of circulating IgA immune complexes. Hepatology (Baltimore, Md.) 1984. PMID: 6693069


  150. Kleinman RE, Harmatz PR, Jacobson LA, Udall JN, Bloch KJ, Walker WA. Passive transplacental immunization: influence on the detection of enteric antigen in the systemic circulation. Pediatric research 1983. PMID: 6877896


  151. Harmatz PR, Kleinman RE, Bunnell BW, Bloch KJ, Walker WA. Hepatobiliary clearance of IgA immune complexes formed in the circulation. Hepatology (Baltimore, Md.) 1982. PMID: 7076115



  152. Seidel JS, Harmatz P, Visvesvara GS, Cohen A, Edwards J, Turner J. Successful treatment of primary amebic meningoencephalitis. The New England journal of medicine 1982. PMID: 7054710



  153. Harmatz P, Boelkins RC, Kessler S. Postisolation aggression and olfactory cues. Behavioral biology 1975. PMID: 1168455