Pompe disease study of walking and symptoms over time
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study follows people with Pompe disease to better understand how the disease progresses and how walking function changes. You may help researchers learn what symptoms and test results matter most, without necessarily receiving a new treatment.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have Pompe disease with a specific gene change called c.-32-13T>G in at least one copy of the GAA gene
- You can walk and your six-minute walk test is over 50 meters
- You are between 18 and 80 years old
- You can sign the study consent form and you have health insurance
- You are not on a breathing machine that is invasive (invasive mechanical ventilation)
- You are able to understand and follow the study instructions
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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