POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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New hope for kids with pompe: enzyme combo trial underway
Disease control OngoingThis study tests a new treatment (cipaglucosidase alfa plus miglustat) for children aged 0 to 18 with late-onset Pompe disease. It aims to check safety, how the drug moves through the body, and its effects. Participants may have received prior enzyme therapy or be new to treatmen…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 26, 2026 07:45 UTC
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Gene therapy trial hopes to treat rare muscle disease
Disease control OngoingThis early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated May 26, 2026 07:40 UTC
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New study tracks babies with pompe gene to learn when to start treatment
Knowledge-focused OngoingThis study follows 20 babies and young children who were found through newborn screening to have a gene change linked to late-onset Pompe disease, but who do not have heart problems. Researchers will track their muscle strength, movement, and other health signs for up to 4.5 year…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 26, 2026 07:40 UTC
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Pompe disease study aims to clear path for gene therapy
Knowledge-focused OngoingThis study looks at people with late-onset Pompe disease to measure antibodies against a common gene therapy carrier (AAV8) and the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine that could help track the disease. No new treatment is given—j…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: NA • Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated May 22, 2026 14:03 UTC