GLYCOGEN STORAGE DISEASE TYPE II
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE II explained in plain language.
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Can early enzyme therapy help babies with pompe disease breathe on their own?
Disease control OngoingThis study follows 16 infants aged 6 months or younger with infantile-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. All receive alglucosidase alfa (Myozyme) as part of their routine care. Researchers track how many survive without needing a brea…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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New enzyme therapy gives hope to babies with rare muscle disease
Disease control OngoingThis study tests a new enzyme replacement therapy called avalglucosidase alfa in babies with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is given through an IV every other week for up to 4 years. …
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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Pompe disease patients get continued enzyme therapy in Long-Term safety study
Disease control OngoingThis study follows 17 people with Pompe disease who have already taken part in earlier trials of avalglucosidase alfa (Nexviadyme). It aims to check the drug's long-term safety and effectiveness by giving it every two weeks as an IV infusion. The study will continue until the dru…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC