INFANTILE-ONSET POMPE DISEASE
Clinical trials for INFANTILE-ONSET POMPE DISEASE explained in plain language.
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Can early enzyme therapy help babies with pompe disease breathe on their own?
Disease control OngoingThis study follows 16 infants aged 6 months or younger with infantile-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. All receive alglucosidase alfa (Myozyme) as part of their routine care. Researchers track how many survive without needing a brea…
Matched conditions: INFANTILE-ONSET POMPE DISEASE
Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 14:03 UTC
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New enzyme therapy gives hope to babies with rare muscle disease
Disease control OngoingThis study tests a new enzyme replacement therapy called avalglucosidase alfa in babies with infantile-onset Pompe disease, a rare genetic disorder that causes severe muscle weakness and breathing problems. The treatment is given through an IV every other week for up to 4 years. …
Matched conditions: INFANTILE-ONSET POMPE DISEASE
Phase: PHASE3 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 27, 2026 12:05 UTC
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New hope for kids with pompe disease: experimental drug shows promise
Disease control OngoingThis 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: INFANTILE-ONSET POMPE DISEASE
Phase: PHASE2 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC