POMPE DISEASE
Clinical trials for POMPE DISEASE explained in plain language.
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First-in-Human gene therapy tested for rare muscle disease
Disease control OngoingThis is an early-stage safety study testing a new gene therapy called ACTUS-101 for adults with late-onset Pompe disease. The therapy aims to deliver a working copy of a missing gene to the liver to help the body produce a needed enzyme. The study will monitor seven participants …
Matched conditions: POMPE DISEASE
Phase: PHASE1 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC
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New test aims to stop dangerous falls for people with Muscle-Weakening diseases
Diagnosis ENROLLING_BY_INVITATIONThis study aims to create and validate a simple set of physical tests to better predict the risk of falling for people with various nerve and muscle disorders. Researchers will test 108 patients with conditions like muscular dystrophy and ALS using tasks like standing up from a c…
Matched conditions: POMPE DISEASE
Sponsor: LMU Klinikum • Aim: Diagnosis
Last updated Mar 20, 2026 14:47 UTC
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Tracking a rare disease from birth: study seeks clues for better future care
Knowledge-focused OngoingThis study aims to learn more about the early signs and symptoms of late-onset Pompe disease in babies and young children who are diagnosed through newborn screening. Researchers will closely monitor 20 children over several years to understand how the disease develops and to hel…
Matched conditions: POMPE DISEASE
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:29 UTC