Gene therapy for late-onset pompe disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene therapy for late-onset Pompe disease. It aims to see if the treatment is safe and effective, and might provide a long-term alternative to enzyme replacement therapy.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 18 years old and have a confirmed diagnosis of late-onset Pompe disease.
- You must have been on enzyme replacement therapy (ERT) before, but have stopped it for at least 4 weeks prior to the study.
- Your breathing must be stable enough that you don't need a ventilator during the day or invasive breathing support.
- Your heart function must be reasonably good (no severe heart failure).
- You should be able to walk at least 100 meters in 6 minutes without using a walker or cane.
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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