Gene therapy for Parkinson's with GBA1 mutation
Part of Brain & nervous system clinical trials.
This study tests a new gene therapy called LY-N001, which is injected directly into the brain. It is for people with moderate to advanced Parkinson's disease who have a specific genetic change in the GBA1 gene. The goal is to see if this treatment is safe and can help improve symptoms.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 30 and 70 years old.
- You have Parkinson's disease with a change in the GBA1 gene.
- Your symptoms have been present for at least 3 years.
- You are in moderate to advanced stages (Hoehn-Yahr stage between 2.5 and 4 when off medication).
- You have not had deep brain stimulation or other brain surgeries.
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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