Testing existing drugs for mitochondrial disorders
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial looks at whether drugs already approved for other conditions might help people with Leigh syndrome, a rare mitochondrial disorder. Researchers will study cells made from your skin to see how these drugs work.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a genetic mutation that causes Leigh syndrome.
- You must have brain MRI changes typical of Leigh syndrome.
- You must not have a bleeding disorder that makes a skin biopsy unsafe.
- You must be willing to provide a small skin sample.
- You must not withdraw your consent to participate.
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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