POMPE DISEASE
Clinical trials for POMPE DISEASE 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
Phase: PHASE4 • Sponsor: Iris Plug • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New test could predict falls in muscle disease patients
Diagnosis ENROLLING_BY_INVITATIONThis study aims to create a simple test battery to determine fall risk in people with neuromuscular disorders, such as muscular dystrophy or ALS. Researchers will assess 108 participants using several physical tests like walking, standing, and rising from a chair. The goal is to …
Matched conditions: POMPE DISEASE
Sponsor: LMU Klinikum • Aim: Diagnosis
Last updated Jun 26, 2026 16:30 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
Sponsor: Duke University • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC