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 a drug called avalglucosidase alfa in 13 Chinese children with infantile-onset Pompe disease, a rare genetic condition that weakens the heart and muscles. The treatment is given as an IV infusion every two weeks for 52 weeks. Researchers will check if it helps ch…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 12, 2026 13:43 UTC
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Global pompe registry aims to unlock secrets of rare disease
Knowledge-focused Recruiting nowThis study is a worldwide registry that collects information from people with Pompe disease, both treated and untreated. The goal is to better understand how the disease progresses and how treatments work over the long term. Anyone with a confirmed diagnosis of Pompe disease can …
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated May 15, 2026 11:54 UTC