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 with rare muscle disease: enzyme therapy trial launches in china
Disease control Recruiting nowThis study tests a drug called avalglucosidase alfa in 13 Chinese children with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and heart problems. Participants will receive the drug through an IV every two weeks for about a year. The goa…
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
-
Global pompe registry aims to unlock secrets of rare disease
Knowledge-focused Recruiting nowThis global registry enrolls people with Pompe disease to track how the condition changes over time, whether they receive treatment or not. By collecting data from up to 2,000 participants, researchers hope to better understand the disease's progression and improve patient care. …
Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:01 UTC