Gene therapy for Best disease retinal trial
Part of Eyes & vision, Genetic & congenital clinical trials.
This trial tests a gene therapy called OPGx-BEST1 injected under the retina to treat vision loss from Best vitelliform macular dystrophy or a related condition. It may help people with advanced disease who have a specific genetic change in the BEST1 gene.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a genetic test showing a disease-causing change in the BEST1 gene.
- You must be 18 years or older.
- Your vision in at least one eye must be between 20/50 and 20/200 for most participants, or 20/200 or worse for the first person in the group.
- You must have active fluid or yellow material under your retina from Best disease.
- You cannot have had prior gene therapy or certain other eye conditions.
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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