MUCOPOLYSACCHARIDOSIS VII
Clinical trials for MUCOPOLYSACCHARIDOSIS VII 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: MUCOPOLYSACCHARIDOSIS 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: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Apr 04, 2026 07:51 UTC
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Massive study launched to map rare disease journey
Knowledge-focused Recruiting nowThis study aims to gather detailed health information from 1000 French patients with a group of rare genetic disorders called Mucopolysaccharidosis (MPS). Researchers will look back at past medical records and follow patients forward to understand how these diseases naturally pro…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:34 UTC