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
-
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 launches to map rare pompe disease journey
Knowledge-focused Recruiting nowThis global registry aims to better understand Pompe disease by tracking its natural progression and treatment outcomes over time. It will enroll 2000 patients worldwide, both treated and untreated, to collect long-term health data. The information will help doctors improve monit…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated Apr 03, 2026 14:42 UTC