POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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One-Shot gene therapy trial offers new hope for debilitating muscle disease
Disease control Recruiting nowThis early-stage study is testing a single infusion of an experimental gene therapy called AB-1009 in adults with late-onset Pompe disease. The goal is to see if this one-time treatment is safe and if it can help control the disease, which causes progressive muscle weakness. Part…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE1, PHASE2 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Apr 01, 2026 23:26 UTC
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Zapping muscles to move: new hope for those who Can't exercise?
Symptom relief Recruiting nowThis study is testing a special type of exercise for people with neuromuscular diseases (like ALS or muscular dystrophy) who often can't do regular workouts. It uses a suit that sends mild electrical signals to make muscles contract, aiming to improve strength, walking, balance, …
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: NA • Sponsor: University of Missouri-Columbia • Aim: Symptom relief
Last updated Mar 30, 2026 14:33 UTC
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Tracking motherhood with a rare muscle disease
Knowledge-focused Recruiting nowThis study aims to collect information about pregnancy and infant development for women with Pompe disease, a rare genetic disorder. It will follow women who are pregnant, whether they are receiving standard enzyme replacement therapy or not, and track their babies' growth for th…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Sponsor: Genzyme, a Sanofi Company • Aim: Knowledge-focused
Last updated Apr 01, 2026 23:26 UTC