GLYCOGEN STORAGE DISEASE TYPE II
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE II explained in plain language.
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New hope for babies with rare muscle disease: enzyme therapy trial launches in china
Disease control Recruiting nowThis study tests an enzyme replacement therapy called avalglucosidase alfa in up to 13 Chinese children with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and heart problems. Participants receive the drug by IV every two weeks for 52 we…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 26, 2026 05:49 UTC
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Global pompe registry opens to all patients to unlock disease secrets
Knowledge-focused Recruiting nowThis study is a worldwide registry that collects information from people with Pompe disease, whether they are receiving treatment or not. The goal is to better understand how the disease progresses and varies among patients. By tracking outcomes over time, researchers hope to imp…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated May 26, 2026 05:55 UTC