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 babies battling rare Muscle-Wasting disease

Disease control Ongoing

This study is testing a new enzyme replacement drug called avalglucosidase alfa for children under 18 with severe infantile-onset Pompe disease. It's for children who are already receiving the current standard treatment but are still getting worse or not responding well enough. T…

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

Phase: PHASE2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control

Last updated Apr 02, 2026 09:12 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 babies battling rare Muscle-Wasting disease