POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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Pompe patients get extended look at Two-Drug Therapy's safety
Disease control CompletedThis study followed 119 adults with late-onset Pompe disease who had already completed an earlier trial. They received an intravenous drug (ATB200) plus an oral pill (AT2221) to see if the combination remained safe and effective over the long term. The main focus was on side effe…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 26, 2026 05:55 UTC
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New drug combo shows promise for pompe disease patients
Disease control CompletedThis study tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles and breathing. The treatment combines an IV drug (ATB200) with a pill (AT2221) to see if it helps patients walk farther and breathe better compared to the curr…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 26, 2026 05:44 UTC