SLY SYNDROME
Clinical trials for SLY SYNDROME 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: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 12:02 UTC
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New study tracks rare disease MPS VII to understand its course and treatment safety
Knowledge-focused Recruiting nowThis study aims to better understand how MPS VII (Sly Syndrome) progresses over time in up to 50 people, both those receiving the drug vestronidase alfa and those not. Researchers will track disease symptoms, biomarkers, and long-term safety, including allergic reactions and immu…
Matched conditions: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 26, 2026 12:44 UTC