Natural history study of RYR1-related disorders
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study follows people with RYR1-related disorders over time to learn how the condition progresses. It may help researchers plan future treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 7 years old.
- You must have a confirmed RYR1 genetic mutation found through standard testing.
- You must live in the United States.
- You must be able to walk 10 meters (about 33 feet) with or without help.
- You cannot be on a breathing machine or have a tracheostomy.
- You cannot have another muscle disease that causes weakness.
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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