MPS VII
Clinical trials for MPS VII explained in plain language.
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Last-Resort drug access opens for rare MPS VII 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 who meet specific criteria. 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 May 14, 2026 12:03 UTC
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Groundbreaking trial aims to treat fatal diseases in the womb
Disease control Recruiting nowThis study tests whether giving enzyme replacement therapy to fetuses with certain lysosomal storage diseases (like MPS disorders, Gaucher, or Pompe) before birth is safe and possible. About 10 pregnant participants will receive the drug through the fetal umbilical vein. The goal…
Matched conditions: MPS VII
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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Scientists launch Long-Term watch on rare sly syndrome
Knowledge-focused Recruiting nowThis study follows up to 50 people with MPS VII (Sly Syndrome) to learn how the disease changes over time and how well the treatment vestronidase alfa works in the long run. Researchers will track symptoms, lab results, and any side effects like allergic reactions. The goal is to…
Matched conditions: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 17, 2026 08:17 UTC