POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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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 4 years to check the long-term safety and effectiveness of a treatment called ATB200 given through an IV along with an oral medication called AT2221. Participants had already completed an earlier study and con…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Apr 03, 2026 14:42 UTC
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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