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TITLE: Long-term outcomes of patients with mucopolysaccharidosis VI treated with galsulfase enzyme replacement therapy since infancy  Full Text
AUTHORS: Garcia, P; Phillips, D; Johnson, J; Martin, K; Randolph, LM; Rosenfeld, H; Harmatz, P;
PUBLISHED: 2021, SOURCE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 133, ISSUE: 1
INDEXED IN: Scopus WOS
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TITLE: Enzyme replacement therapy in patients with mucopolysaccharidosis type VI: Updated findings from the MPS VI clinical surveillance program  Full Text
AUTHORS: Paul Harmatz; Christina Lampe; Rossella Parini; Reena Sharma; Elisa Leao Teles; Julie Johnson; Debbie Sivam; Zlatko Sisic;
PUBLISHED: 2019, SOURCE: 15th Annual Research Meeting of the WORLDSymposium(TM) in MOLECULAR GENETICS AND METABOLISM, VOLUME: 126, ISSUE: 2
INDEXED IN: WOS
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TITLE: Mucopolysaccharidosis type VI enzyme replacement therapy outcomes across the disease spectrum: findings from the MPS VI clinical surveillance program  Full Text
AUTHORS: Paul Harmatz; Christina Lampe; Rossella Parini; Reena Sharma; Elisa Ledo Teles; Sara Hawley; Julie Johnson; Debbie Sivam; Zlatko Sisic;
PUBLISHED: 2018, SOURCE: We're Organizing Research for Lysosomal Diseases (WORLD) Symposium in MOLECULAR GENETICS AND METABOLISM, VOLUME: 123, ISSUE: 2
INDEXED IN: WOS
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TITLE: Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program  Full Text
AUTHORS: Paul Harmatz; Christina Lampe; Rossella Parini; Reena Sharma; Elisa Lea Teles; Sara Hawley; Julie Johnson; Debbie Sivam; Zlatko Sisic;
PUBLISHED: 2018, SOURCE: We're Organizing Research for Lysosomal Diseases (WORLD) Symposium in MOLECULAR GENETICS AND METABOLISM, VOLUME: 123, ISSUE: 2
INDEXED IN: WOS
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TITLE: MUCOPOLYSACCHARIDOSIS VI ENZYME REPLACEMENT THERAPY OUTCOMES ACROSS THE DISEASE SPECTRUM: FINDINGS FROM THE MPS VI CLINICAL SURVEILLANCE PROGRAM  Full Text
AUTHORS: Harmatz, P; Lampe, C; Parini, R; Sharma, R; Leao Teles, EL; Hawley, S; Johnson, J; Sivam, D; Sisic, Z;
PUBLISHED: 2018, SOURCE: 40th Annual Meeting of the Society-for-Inherited-Metabolic-Disorders (SIMD) in MOLECULAR GENETICS AND METABOLISM, VOLUME: 123, ISSUE: 3
INDEXED IN: WOS
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TITLE: MUCOPOLYSACCHARIDOSIS VI ENZYME REPLACEMENT THERAPY INITIATED IN ADULTHOOD: FINDINGS FROM THE MPS VI CLINICAL SURVEILLANCE PROGRAM  Full Text
AUTHORS: Lampe, C; Harmatz, P; Parini, R; Sharma, R; Leao Teles, EL; Hawley, S; Johnson, J; Sivam, D; Sisic, Z;
PUBLISHED: 2018, SOURCE: 40th Annual Meeting of the Society-for-Inherited-Metabolic-Disorders (SIMD) in MOLECULAR GENETICS AND METABOLISM, VOLUME: 123, ISSUE: 3
INDEXED IN: WOS
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TITLE: The effect of galsulfase enzyme replacement therapy on the growth of patients with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)  Full Text
AUTHORS: Harmatz, P; Hendriksz, CJ; Lampe, C; McGill, JJ; Parini, R; Leao Teles, E; Valayannopoulos, V; Tj. Cole; Matousek, R; Graham, S; Guffon, N; Quartel, A;
PUBLISHED: 2017, SOURCE: MOLECULAR GENETICS AND METABOLISM, VOLUME: 122, ISSUE: 1-2
INDEXED IN: WOS
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TITLE: Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial  Full Text
AUTHORS: 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; Ben Omran, T; Bober, MB; Burton, BK; Cleary, MA; Dali, CI; de Medeiros, PFV; Guelbert, NB; Hiwot, TG; Jin, DK; Jones, SA; Lin, SP; Maranda, B; Mitchell, JJ; Murphy, EM; Muschol, N; Okuyama, T; Santra, S; Villarreal, MLS; Steiner, R; Tanpaiboon, P; Valente, PC; Matos, SBG; Vellodi, A; White, KK; Wijburg, FA; Yang, K; Mealiffe, M; Haller, C; ...More
PUBLISHED: 2015, SOURCE: Molecular Genetics and Metabolism, VOLUME: 114, ISSUE: 2
INDEXED IN: Scopus CrossRef
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TITLE: Galsulfase (NaglazymeA (R)) therapy in infants with mucopolysaccharidosis VI  Full Text
AUTHORS: Paul R Harmatz; Paula Garcia; Nathalie Guffon; Linda M Randolph; Renee Shediac; Elizabeth Braunlin; Ralph S Lachman; Celeste Decker;
PUBLISHED: 2014, SOURCE: JOURNAL OF INHERITED METABOLIC DISEASE, VOLUME: 37, ISSUE: 2
INDEXED IN: WOS CrossRef
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TITLE: 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  Full Text
AUTHORS: Roberto Giugliani; Christina Lampe; Nathalie Guffon; David Ketteridge; Elisa Leao Teles; James E Wraith; Simon A Jones; Cheri Piscia Nichols; Ping Lin; Adrian Quartel; Paul Harmatz;
PUBLISHED: 2014, SOURCE: AMERICAN JOURNAL OF MEDICAL GENETICS PART A, VOLUME: 164A, ISSUE: 8
INDEXED IN: Scopus WOS CrossRef
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