INFANTILE-ONSET POMPE DISEASE
Clinical trials for INFANTILE-ONSET POMPE DISEASE explained in plain language.
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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: INFANTILE-ONSET POMPE DISEASE
Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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Pompe disease patients invited to join worldwide registry
Knowledge-focused Recruiting nowThis study is a global registry that will follow about 500 people with Pompe disease over time. It includes both treated and untreated patients, and aims to collect real-world data on treatment safety, effectiveness, and quality of life. No new treatments are being tested—this is…
Matched conditions: INFANTILE-ONSET POMPE DISEASE
Sponsor: Amicus Therapeutics • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC