Gene therapy trial for children with BBS1 retinal degeneration
Part of Eyes & vision clinical trials.
This early-phase trial tests a gene therapy called AXV-101 for children with vision loss caused by BBS1 genetic mutations. The goal is to see if it can help slow or stop retinal damage.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Aged 4 to 17 years old.
- Must have a confirmed BBS1 genetic mutation (two copies, one from each parent).
- Show signs of retinal degeneration, like trouble seeing at night or cone-rod dystrophy.
- Have enough healthy retinal cells measured by a special eye scan (OCT).
- Parent or guardian must agree to the trial and follow study visits.
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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