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. Mepsevii is an enzyme replacement therapy designed to help manage the disease. Patients must meet specific eligibility criteria to be considered.
Matched conditions: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 09:50 UTC
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Groundbreaking trial aims to treat fatal diseases in the womb
Disease control Recruiting nowThis early-stage study tests whether giving enzyme replacement therapy to fetuses with certain genetic disorders (like MPS and Pompe disease) is safe and feasible. About 10 pregnant participants will receive the drug through the umbilical vein. The goal is to see if this approach…
Matched conditions: MPS VII
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 26, 2026 09:48 UTC
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Scientists track rare disease to improve care
Knowledge-focused Recruiting nowThis study follows up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. Researchers will also check how well and how safely the drug vestronidase alfa works in the long run. Participants may or may not be taking this treatment. The goal is …
Matched conditions: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 20, 2026 11:54 UTC