Personalized gene therapy for ALS linked to TARDBP
Part of Brain & nervous system, Hormones & metabolism clinical trials.
This trial tests a personalized medicine approach for people with ALS caused by changes in the TARDBP gene. The treatment uses a custom-made molecule to target the genetic cause and may help slow the disease.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed genetic diagnosis of a neurological disorder (likely ALS) caused by a TARDBP gene change.
- You are able to travel to the study site for visits and follow-ups.
- You are willing to provide access to your medical records.
- You have not used an investigational drug within a certain time before starting (less than 5 half-lives).
- You have no other condition that, in the doctor's opinion, would prevent you from completing the study.
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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