Mucopolysaccharidosis Type Vi
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CLINICAL_TRIALS other · 2026-04-08Clinical Trial Update: Registry of Patients Diagnosed With Lysosomal Storage Diseases
This ClinicalTrials.gov registry entry describes ongoing recruitment for a registry of patients diagnosed with various lysosomal storage dis…
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) [Phase 1]
Phase 1 prenatal enzyme replacement therapy trial PEARL is currently recruiting for multiple lysosomal storage disorders including MPS I, MP…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: MPS (RaDiCo Cohort) (RaDiCo-MPS)
An update on the RaDiCo-MPS clinical trial cohort sponsored by INSERM, recruiting patients with several mucopolysaccharidosis types I-IX and…
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CLINICAL_TRIALS research · 2026-02-02Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI [Phase 1, Phase 2]
A clinical trial is evaluating the safety and efficacy of adalimumab in mucopolysaccharidosis types I, II, and VI; the study is currently ac…
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EMA approval · 2024-12-16EMA Authorised: Naglazyme (galsulfase) — BioMarin International Limited
The European Medicines Agency authorised Naglazyme (galsulfase) by BioMarin International Limited for treating mucopolysaccharidosis type VI…
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CLINICAL_TRIALS trial_result · 2024-11-22Clinical Trial Update: Gene Therapy in Patients With Mucopolysaccharidosis Disease [Phase 1, Phase 2]
This clinical trial update reports completion of a Phase 1/2 gene therapy study in patients with Mucopolysaccharidosis Type VI. The status o…
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CLINICAL_TRIALS trial_result · 2024-10-22Clinical Trial Update: Longitudinal Study of Bone Disease in Children with Mucopolysaccharidoses (MPS) I, II, and VI
This clinical trial update reports on a longitudinal study of bone disease in children with mucopolysaccharidoses types I, II, and VI. The s…
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FDA approval · 2019-12-17FDA Approved: NAGLAZYME — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved a supplemental application for Naglazyme (galsulfase) from BioMarin Pharmaceutical Inc. The notice indicates regulatory approva…
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CLINICAL_TRIALS trial_result · 2017-09-15Clinical Trial Update: Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI [Phase 1, Phase 2]
Clinical trial evaluating adalimumab in mucopolysaccharidosis types I, II, and VI has completed phases 1 and 2 with results posted in 2017.
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CLINICAL_TRIALS research · 2015-08-04Clinical Trial Update: Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
A completed clinical trial evaluating diagnostic approaches for mucopolysaccharidosis type IV A and type VI in patients presenting with bila…
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CLINICAL_TRIALS trial_result · 2014-04-02Clinical Trial Update: Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02
This is a completed observational study of patients with Mucopolysaccharidosis type VI who previously participated in ASB-00-02, sponsored b…
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FDA approval · 2013-03-26FDA Approved: NAGLAZYME — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for Naglazyme (galsulfase) from BioMarin Pharmaceutical Inc.
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FDA approval · 2011-04-28FDA Approved: NAGLAZYME — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental Biologics License Application for Naglazyme (galsulfase) by BioMarin Pharmaceutical Inc., updating the label…
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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…
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CLINICAL_TRIALS trial_result · 2009-08-25Clinical Trial Update: Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI [Phase 2]
Open-label Phase 2 study evaluating the efficacy and safety of Naglazyme (recombinant human N-acetylgalactosamine 4-sulfatase) in patients w…
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CLINICAL_TRIALS trial_result · 2009-08-17Clinical Trial Update: Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI [Phase 3]
A Phase 3 trial of recombinant human N-acetylgalactosamine 4-sulfatase (galsulfase) for mucopolysaccharidosis type VI has completed and resu…