POMPE DISEASE
Clinical trials for POMPE DISEASE explained in plain language.
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Gene therapy hope for pompe patients in early safety trial
Disease control CompletedThis early-phase study tested a new gene therapy called ACTUS-101 in 7 adults with late-onset Pompe disease. The main goal was to check safety and side effects. Participants received a single intravenous dose, and researchers monitored their health over time.
Matched conditions: POMPE DISEASE
Phase: PHASE1 • Sponsor: AskBio Inc • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC
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New drug combo shows promise for rare pompe disease
Disease control CompletedThis study tested whether a new combination of two drugs (ATB200 and AT2221) is safe for adults with Pompe disease, a rare genetic disorder that causes muscle weakness. 29 participants received the drugs through an IV and by mouth. The main goal was to check for side effects and …
Matched conditions: POMPE DISEASE
Phase: PHASE1, PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:55 UTC
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Heart condition costs under the microscope in massive hospital study
Knowledge-focused CompletedThis study collected information from over 15,000 people hospitalized with cardiomyopathy, a heart muscle disease. Researchers looked at how much these hospital stays cost and what the money was spent on. The goal was to understand the real-world financial impact and help manage …
Matched conditions: POMPE DISEASE
Sponsor: China National Center for Cardiovascular Diseases • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:35 UTC
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Can a breath test reveal how muscles make energy? tiny study explores new way to track treatment effects
Knowledge-focused CompletedThis small pilot study looked at whether simple breath and urine tests can measure how well muscles produce energy in children with metabolic myopathies (rare muscle disorders). Three participants completed a 12-week at-home physiotherapy program. The goal was to see if these non…
Matched conditions: POMPE DISEASE
Phase: NA • Sponsor: University of British Columbia • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:02 UTC