Val-1221 for lafora disease expanded access
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial gives an experimental treatment called VAL-1221 to people with Lafora disease, a rare genetic condition that causes seizures and muscle problems. The goal is to slow down the disease and improve quality of life.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed genetic test showing changes (mutations) in the EPM2A or EPM2B gene that cause Lafora disease.
- You should be in the middle phase of the disease, meaning you are between 12 and 28 years old.
- You need a committed helper (like a family member) who is at least 18 years old and can support you throughout the trial.
- You must be willing to travel to the trial site for study visits and procedures.
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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