Maroteaux-lamy Syndrome
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-03-10Clinical Trial Update: Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VI [Phase 1, Phase 2]
This clinical trial update reports safety and efficacy data for Voxzogo (vosoritide) in growth deficits associated with Morquio A syndrome (…
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CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
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CLINICAL_TRIALS trial_result · 2020-12-23Clinical Trial Update: Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome [NA]
A clinical trial update for Naglazyme after allo-transplant in Maroteaux-Lamy Syndrome has been terminated, with results posted in 2020.
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CLINICAL_TRIALS trial_result · 2015-08-13Clinical Trial Update: Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
This completed trial update describes screening an orthopedic population for mildly affected individuals with Morquio syndrome type A and Ma…
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CLINICAL_TRIALS trial_result · 2011-07-13Clinical Trial Update: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism [Phase 2]
Phase 2 trial update on hematopoietic stem cell transplantation for inborn errors of metabolism; trial terminated with results posted in 201…
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CLINICAL_TRIALS trial_result · 2010-10-15Clinical Trial Update: A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI [Phase 4]
This clinical trial update reports Phase 4 results from a two-dose level study of Naglazyme (galsulfase) in infants with Mucopolysaccharidos…