POMPE DISEASE
Clinical trials for POMPE DISEASE explained in plain language.
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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 17, 2026 04:15 UTC
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Gene therapy hope for pompe disease patients in early safety trial
Disease control CompletedThis early-stage study tested the safety of a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The treatment aims to help the body produce an enzyme it lacks, potentially reducing the need for regular infusions. The study focused on side effects and la…
Matched conditions: POMPE DISEASE
Phase: PHASE1 • Sponsor: AskBio Inc • Aim: Disease control
Last updated May 15, 2026 11:56 UTC