POMPE DISEASE
Clinical trials for POMPE DISEASE explained in plain language.
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New gene therapy shows promise for rare muscle disease
Disease control CompletedThis early-stage study tested a gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease, a condition that causes muscle weakness. The main goal was to check safety and see if the treatment could increase the missing enzyme. Participants received a single intraveno…
Matched conditions: POMPE DISEASE
Phase: PHASE1 • Sponsor: AskBio Inc • Aim: Disease control
Last updated May 26, 2026 08:19 UTC
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New drug combo shows promise for rare pompe disease
Disease control CompletedThis study tested two experimental drugs, ATB200 and AT2221, given together to adults with Pompe disease. The goal was to see if the combination is safe and how the body handles it. 29 participants received the drugs through an IV and a pill. The study found that the treatment wa…
Matched conditions: POMPE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 18, 2026 11:59 UTC