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 10:41 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: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 20, 2026 11:54 UTC