Study of UX016 for GNE myopathy (GNEM)
Part of Bones, joints & muscles, Genetic & congenital clinical trials.
This trial tests a new medicine called UX016 for people with GNE myopathy, a rare muscle-weakening disease. The goal is to see if it can help improve muscle strength and function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of GNE myopathy through genetic testing.
- You must be able to walk at least 20 meters (about 20 steps) on your own, with or without a cane or walker.
- You must have some weakness in your upper arm (biceps) but still be able to use it during strength tests.
- You must be willing to have two small needle biopsies of your thigh muscle during the study.
- You (or your partner) must not be pregnant or planning to become pregnant during the study, and you must use effective birth control.
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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