Gene therapy for late-onset Pompe disease after ERT
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This early-stage trial tests a new gene therapy for adults with late-onset Pompe disease who have already tried enzyme replacement therapy (ERT). The goal is to see if the gene therapy is safe and can help improve muscle strength and breathing.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 18 years old.
- You must have been diagnosed with late-onset Pompe disease (LOPD).
- You must have previously received enzyme replacement therapy (ERT) and stopped it for at least 4 weeks before joining.
- You must be able to walk 100 meters (about the length of a football field) without stopping, and 40 meters without using a cane or walker.
- You must not need a breathing machine during the day, and your heart function must be stable.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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