Early access treatment for FUS-related ALS
Part of Brain & nervous system, Hormones & metabolism clinical trials.
This program offers an experimental drug called ulefnersen to people with ALS caused by a change in the FUS gene. It aims to slow the disease, and it involves regular injections into the fluid around the spinal cord.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed change in the FUS gene that causes ALS.
- You are 10 years or older and have signs of motor nerve loss.
- Your lung function is at least 30% of expected.
- You can safely undergo repeated spinal taps (lumbar punctures).
- Your medications and nutrition have been stable for at least 1 month.
- If you can become pregnant, you must use effective birth control during and after the program.
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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