GLYCOGEN STORAGE DISEASE TYPE II
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE II explained in plain language.
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New enzyme therapy shows promise for infants with rare muscle disease
Disease control OngoingThis study tests a new enzyme replacement therapy called avalglucosidase alfa in infants with Pompe disease, a rare genetic disorder that causes severe muscle weakness. The treatment is given by IV every other week for up to 4 years. The main goal is to see if more babies survive…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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French study tracks Long-Term effects of rare muscle disease treatment
Disease control OngoingThis study continues to provide and monitor an enzyme replacement therapy called avalglucosidase alfa for 17 patients in France with Pompe disease, a rare genetic disorder that weakens muscles. The purpose is to collect long-term safety data and see how well the treatment continu…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Apr 20, 2026 16:15 UTC