POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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New hope for pompe disease: advanced Two-Drug therapy tested in major trial
Disease control CompletedThis study tested a new treatment for adults with late-onset Pompe disease, a rare genetic disorder that weakens muscles. The treatment combines an intravenous drug (ATB200) with an oral pill (AT2221). Researchers compared this new combination to the standard enzyme replacement t…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Mar 30, 2026 14:28 UTC
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Four-Year study tests promising treatment for rare Muscle-Weakening disease
Disease control CompletedThis study followed 119 adults with late-onset Pompe disease for up to four years to see how safe and effective the treatment ATB200/AT2221 is over the long term. Participants had already completed an earlier study and received regular intravenous infusions plus oral medication. …
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Mar 18, 2026 14:41 UTC