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. It is designed for individual patients who meet specific criteria. The goal is to offer a potential treatment when no alternatives exist.
Matched conditions: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 14, 2026 12:03 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: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 17, 2026 07:23 UTC