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.
By submitting, you agree to our Terms of use
-
New hope for babies battling rare, Life-Threatening muscle disease in china
Disease control Recruiting nowThis study is testing the safety and effectiveness of a drug called avalglucosidase alfa for Chinese children under 18 with infantile-onset Pompe disease, a severe genetic disorder. Participants will receive the drug by IV infusion once every two weeks for one year. The main goal…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
-
Global effort launched to map rare muscle disease
Knowledge-focused Recruiting nowThis global registry aims to better understand Pompe disease, a rare genetic disorder that causes muscle weakness. It will track 2,000 patients over time, both those receiving treatment and those who are not, to learn how the disease progresses and how treatments work long-term. …
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated Mar 17, 2026 13:10 UTC