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 pompe disease
Disease control OngoingThis early-stage study is testing a one-time gene therapy called AT845 for adults with late-onset Pompe disease. The goal is to see if delivering a working copy of the faulty gene directly into the body is safe and can help control the disease. Participants receive a single infus…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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New hope for kids with rare Muscle-Weakening disease
Disease control OngoingThis study is testing a new combination drug (cipaglucosidase alfa with miglustat) for children with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The main goal is to see if the treatment is safe and how well it works in children from birth up to 18 year…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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Scientists map immune barriers for future pompe disease gene therapy
Knowledge-focused OngoingThis study aims to gather information that could help develop future gene therapies for late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Researchers are measuring antibody levels against a common virus used in gene therapy (AAV8) and against …
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: NA • Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:35 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 (LATE-ONSET)
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:29 UTC