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. Mepsevii is an enzyme replacement therapy designed to help manage the disease. Patients must meet specific eligibility criteria to be considered.
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Disease control
Last updated May 26, 2026 08:23 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects health information from up to 250 people with lysosomal storage diseases, including pregnant women whose fetus has a diagnosis. Researchers will track symptoms, lab results, and development over time to better understand how these rare conditions progress. The…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 26, 2026 08:30 UTC
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French study aims to map rare MPS diseases in 1000 patients
Knowledge-focused Recruiting nowThis study is collecting health information from 1000 people in France with mucopolysaccharidosis (MPS), a group of rare genetic disorders. Researchers will look at medical records and test results to better understand how the diseases progress and how current treatments affect p…
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France • Aim: Knowledge-focused
Last updated May 26, 2026 08:26 UTC
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Scientists track rare disease to improve care
Knowledge-focused Recruiting nowThis study follows up to 50 people with MPS VII (Sly Syndrome) to understand how the disease changes over time. Researchers will also check how well and how safely the drug vestronidase alfa works in the long run. Participants may or may not be taking this treatment. The goal is …
Matched conditions: MUCOPOLYSACCHARIDOSIS VII
Sponsor: Ultragenyx Pharmaceutical Inc • Aim: Knowledge-focused
Last updated May 20, 2026 11:54 UTC