SLY SYNDROME
Clinical trials for SLY SYNDROME explained in plain language.
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Lifeline offered for rare genetic disorder patients
Disease control AVAILABLEThis program provides access to the drug Mepsevii for patients with a rare genetic disorder called MPS VII (Sly Syndrome). It is for individuals who have no other treatment options available. The goal is to manage the disease by providing the approved therapy in countries where i…
Matched conditions: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
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Global effort launched to map rare genetic disease
Knowledge-focused Recruiting nowThis study aims to better understand how MPS VII (Sly Syndrome) progresses and how the treatment vestronidase alfa works over the long term. It will follow about 50 patients, both those receiving the treatment and those who are not, to collect detailed health information. The goa…
Matched conditions: SLY SYNDROME
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Mar 16, 2026 15:26 UTC