GLYCOGEN STORAGE DISEASE TYPE II
Clinical trials for GLYCOGEN STORAGE DISEASE TYPE II explained in plain language.
Never miss a new study
Get alerted when new GLYCOGEN STORAGE DISEASE TYPE II trials appear
Sign up with your email to follow new studies for GLYCOGEN STORAGE DISEASE TYPE II, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
Pompe disease patients get continued access to enzyme therapy in french study
Disease control OngoingThis study gives Pompe disease patients in France continued access to the enzyme replacement therapy avalglucosidase alfa after they finished earlier clinical trials. It monitors long-term safety and how well the treatment works over time. About 17 adults and children with late-o…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated May 22, 2026 13:53 UTC
-
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