Glycogen storage disease type II-Pompe's disease

Clinical trials for Glycogen storage disease type II-Pompe's disease explained in plain language.

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New hope for kids with pompe disease: experimental drug shows promise

Disease control Ongoing

This phase 2 trial tests a new enzyme replacement therapy called avalglucosidase alfa (Nexviazyme) in 22 children with infantile-onset Pompe disease who are not responding well to standard treatment. The study aims to see if the new drug is safe and can improve outcomes. Particip…

Matched conditions: GLYCOGEN STORAGE DISEASE TYPE II-POMPE'S DISEASE

Phase 2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control

Last updated Jun 27, 2026 09:00 UTC

Glycogen storage disease type II-Pompe's disease

Get alerted when new Glycogen storage disease type II-Pompe's disease trials appear

New hope for kids with pompe disease: experimental drug shows promise