POMPE DISEASE LATE-ONSET
Clinical trials for POMPE DISEASE LATE-ONSET explained in plain language.
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Pompe disease drug combo shows Long-Term safety in phase 3 trial
Disease control CompletedThis study tested the long-term safety of a combination therapy (ATB200 and AT2221) in 119 adults with late-onset Pompe disease who had completed a prior study. Participants received the drugs for an extended period, and researchers tracked side effects, walking distance, and lun…
Matched conditions: POMPE DISEASE LATE-ONSET
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New drug cocktail aims to boost walking and breathing in pompe patients
Disease control CompletedThis phase 3 trial tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. Participants received either the experimental combo (cipaglucosidase alfa plus miglustat) or the current standard therapy (alglucosidase…
Matched conditions: POMPE DISEASE LATE-ONSET
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 16:41 UTC