Mucopolysaccharidosis Type Vii

• CLINICAL_TRIALS other · 2026-04-08
  Clinical 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…

• CLINICAL_TRIALS research · 2026-03-17
  Clinical 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…

• CLINICAL_TRIALS research · 2026-02-11
  Clinical 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…

• FDA approval · 2020-12-23
  FDA Approved: MEPSEVII — Supplemental Application (Ultragenyx Pharmaceutical Inc.)

  The FDA approved Ultragenyx's supplemental application for MEPSEVII (vestronidase alfa), approving updates to its labeling for the treatment…

• FDA approval · 2019-12-06
  FDA Approved: MEPSEVII — Supplemental Application (Ultragenyx Pharmaceutical Inc.)

  The FDA approved a supplemental application for Mepsevii (vestronidase alfa) submitted by Ultragenyx Pharmaceutical Inc.

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• CLINICAL_TRIALS trial_result · 2018-02-19
  Clinical Trial Update: A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) [Phase 3]

  Phase 3 study of UX003 rhGUS enzyme replacement therapy for MPS type 7 (Sly syndrome) completed with results posted in 2018.

• FDA approval · 2017-11-15
  FDA Approved: MEPSEVII — Original Application (Ultragenyx Pharmaceutical Inc.)

  The FDA approved Mepsevii (vestronidase alfa) as an original Biologics License Application for the treatment of mucopolysaccharidosis type V…