POMPE DISEASE LATE-ONSET
Clinical trials for POMPE DISEASE LATE-ONSET explained in plain language.
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Hope for pompe patients: could a switch in enzyme therapy slow decline?
Disease control OngoingThis study tests whether switching to avalglucosidase alfa is safe and more effective for people with late-onset Pompe disease whose condition has worsened on the standard treatment, alglucosidase alfa. Participants receive biweekly infusions of the new drug and are monitored for…
Matched conditions: POMPE DISEASE LATE-ONSET
Phase: PHASE4 • Sponsor: Iris Plug • Aim: Disease control
Last updated Jun 27, 2026 14:00 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 Jun 27, 2026 13:07 UTC
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New hope for kids with pompe: enzyme combo trial underway
Disease control OngoingThis study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least…
Matched conditions: POMPE DISEASE LATE-ONSET
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:55 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 virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years…
Matched conditions: POMPE DISEASE LATE-ONSET
Phase: NA • Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:06 UTC
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New study tracks early signs of pompe disease in newborns
Knowledge-focused OngoingThis study follows 20 newborns and children diagnosed with late-onset Pompe disease through newborn screening. Researchers will track their health for up to 4.5 years to document early muscle and joint symptoms, biomarkers, and quality of life. The goal is to learn when to start …
Matched conditions: POMPE DISEASE LATE-ONSET
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC