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. The therapy aims to help your body produce the enzyme needed to break down glycogen, which may improve muscle and breathing function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 6 years old or older.
- You have been diagnosed with late-onset Pompe disease (LOPD).
- Your lung function test (FVC) is at least 30% of what is expected for your age and size.
- You can walk at least 40 meters in 6 minutes (with or without a walking aid).
- You have not taken part in a gene therapy trial before.
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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