Mucopolysaccharidosis Vii

• EMA approval · 2026-07-08
  EMA Authorised: Mepsevii (vestronidase alfa) — Ultragenyx Germany GmbH

  EMA authorises Mepsevii (vestronidase alfa) for mucopolysaccharidosis VII in the EU.

• CLINICAL_TRIALS research · 2026-04-15
  Clinical Trial Update: Mucopolysaccharidosis VII Disease Monitoring Program

  Ultragenyx Pharmaceutical is running a disease monitoring program for Mucopolysaccharidosis VII that is currently recruiting participants.

• CLINICAL_TRIALS trial_result · 2026-04-07
  Clinical Trial Update: Expanded Access to Mepsevii

  Expanded access program for Mepsevii (vestronidase alfa) for patients with mucopolysaccharidosis VII is currently available.

• EMA approval · 2024-06-28
  EMA Authorised: Mepsevii (vestronidase alfa) — Ultragenyx Germany GmbH

  The European Medicines Agency authorised Mepsevii (vestronidase alfa) from Ultragenyx Germany GmbH for mucopolysaccharidosis VII in the Euro…

• CLINICAL_TRIALS trial_result · 2019-10-16
  Clinical Trial Update: Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age [Phase 2]

  Phase 2 clinical trial evaluating UX003 rhGUS enzyme replacement for MPS VII (Sly Syndrome) in children under 5; study completed with result…

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• CLINICAL_TRIALS trial_result · 2019-07-30
  Clinical Trial Update: A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) [Phase 3]

  Phase 3 trial evaluating UX003 rhGUS for MPS VII (Sly syndrome) has completed; results posted in 2019.