MPS VII
Clinical trials for MPS VII explained in plain language.
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Last-Resort drug access opens for rare sly syndrome patients
Disease control AVAILABLEThis program provides expanded access to Mepsevii for people with MPS VII (Sly Syndrome) who have no other treatment options. It is designed for individual patients on a case-by-case basis. The goal is to offer a potential treatment when no alternatives exist.
Matched conditions: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Groundbreaking trial aims to treat rare diseases in the womb
Disease control Recruiting nowThis study tests whether giving enzyme replacement therapy to fetuses with certain rare genetic diseases (like MPS I, Gaucher, or Pompe) before birth is safe and feasible. About 10 pregnant participants will receive the treatment through the umbilical vein. The goal is to see if …
Matched conditions: MPS VII
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Rare disease study tracks MPS VII over time
Knowledge-focused Recruiting nowThis study monitors up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. It also checks the long-term safety and effectiveness of the drug vestronidase alfa. Participants may or may not be taking the drug, and the study involves regular che…
Matched conditions: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:07 UTC