MPS VII
Clinical trials for MPS VII explained in plain language.
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Doctors test Life-Saving enzyme treatment for babies before they are born
Disease control Recruiting nowThis early-stage study is testing whether giving enzyme replacement therapy to fetuses still in the womb is safe and feasible. It aims to treat rare, severe genetic disorders that often cause life-threatening complications before or shortly after birth. The hope is that treating …
Matched conditions: MPS VII
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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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: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
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Global study tracks rare disease to understand treatment impact
Knowledge-focused Recruiting nowThis study aims to better understand how MPS VII (Sly Syndrome) progresses over time and to gather long-term information on the effectiveness and safety of the treatment vestronidase alfa. It will follow up to 50 patients, both those receiving the treatment and those who are not,…
Matched conditions: MPS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Apr 04, 2026 03:36 UTC