Surlorian for RYR1 muscle weakness: strength and safety study
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This trial tests whether a new drug called Surlorian can improve muscle strength in adults with a rare genetic muscle condition (RYR1-related myopathy). You'd receive either the drug or a placebo and be monitored for safety and changes in muscle function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic diagnosis of RYR1-related myopathy with an autosomal dominant mutation
- You experience weakness in upper arm, shoulder, or hip muscles (proximal muscles)
- You can walk 10 meters, with or without a cane
- You are willing to use effective birth control if you can have children, or you cannot have children
- You do not have severe lung disease, heart disease, or other neuromuscular conditions besides RYR1-myopathy
- You are not currently taking certain medications (like statins, acid reflux drugs, or specific heart/liver medications) or can stop them before starting
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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