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 combination of two medicines (cipaglucosidase alfa and miglustat) in children aged 0 to 18 with late-onset Pompe disease, a rare genetic disorder that causes muscle weakness. The goal is to see if the treatment is safe and works better than current enzyme repla…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 17, 2026 04:32 UTC
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Gene therapy could free pompe patients from weekly infusions
Disease control OngoingThis study tests a one-time gene therapy called AT845 for adults with late-onset Pompe disease. The goal is to help the body produce the missing enzyme on its own, potentially reducing or replacing the need for regular enzyme infusions. The trial is small (11 participants) and fo…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE1, PHASE2 • Sponsor: Astellas Gene Therapies • Aim: Disease control
Last updated May 15, 2026 11:53 UTC
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New study tracks early signs of pompe in screened newborns
Knowledge-focused OngoingThis study follows 20 newborns and children who were found to have late-onset Pompe disease through newborn screening. Researchers track muscle and joint symptoms, lab results, and quality of life over several years to learn how the disease develops. No treatments are given; the …
Matched conditions: POMPE DISEASE (LATE-ONSET)
Sponsor: Duke University • Aim: Knowledge-focused
Last updated May 15, 2026 11:57 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 that might interfere with future gene therapy. Participants are either new to enzyme replacement therapy or have been on it for at least six months. No treatment is given; instead, blood and urine samp…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: NA • Sponsor: Astellas Gene Therapies • Aim: Knowledge-focused
Last updated May 15, 2026 11:55 UTC