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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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
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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: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated Mar 16, 2026 15:26 UTC