POMPE DISEASE (LATE-ONSET)
Clinical trials for POMPE DISEASE (LATE-ONSET) explained in plain language.
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New pompe combo therapy shows promise in walking and breathing tests
Disease control CompletedThis study tested a new treatment for adults with late-onset Pompe disease, a rare muscle-weakening condition. 125 participants received either the experimental drug combo (ATB200 plus AT2221) or the current standard treatment. Researchers measured how far people could walk in 6 …
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 11, 2026 20:51 UTC
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New hope for pompe patients: Long-Term drug combo shows promise
Disease control CompletedThis study looked at the long-term safety and effectiveness of a treatment combining two drugs (ATB200 and AT2221) for adults with late-onset Pompe disease, a rare genetic disorder that causes muscle weakness. All 119 participants had already completed an earlier study of the sam…
Matched conditions: POMPE DISEASE (LATE-ONSET)
Phase: PHASE3 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 11, 2026 20:39 UTC