MUCOPOLYSACCHARIDOSIS VII
Clinical trials for MUCOPOLYSACCHARIDOSIS VII 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: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 14, 2026 12:03 UTC
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New registry aims to unlock secrets of rare lysosomal diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 people with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to learn how these conditions develop and how they appear before birth. Researchers will review medical records and test samples t…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 17, 2026 07:25 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: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 17, 2026 07:23 UTC
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French researchers launch largest-ever MPS patient registry to unlock disease secrets
Knowledge-focused Recruiting nowThis study will collect health information from 1,000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers aim to better understand how the disease progresses and how it is currently managed with or without specific treatments. No new …
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated Apr 29, 2026 15:03 UTC