POMPE'S DISEASE

Clinical trials for POMPE'S DISEASE explained in plain language.

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Trials to join now! 1

New hope for babies with rare muscle disease: enzyme therapy trial launches in china

Disease control Recruiting now

This study tests a drug called avalglucosidase alfa in 13 Chinese children with infantile-onset Pompe disease, a rare genetic condition that weakens the heart and muscles. The treatment is given as an IV infusion every two weeks for 52 weeks. Researchers will check if it helps ch…

Matched conditions: POMPE'S DISEASE

Phase: PHASE4 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control

Last updated May 12, 2026 13:43 UTC

POMPE'S DISEASE

Get alerted when new POMPE'S DISEASE trials appear

New hope for babies with rare muscle disease: enzyme therapy trial launches in china