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BEST1-related dominant retinopathy

MONDO:0700238

Any retinopathy caused by a heterozygous variant in the BEST1 gene.

Also known as: BEST1-related dominant retinopathy

31 clinical trials for this condition and its sub-types, 1 tagged with BEST1-related dominant retinopathy itself.

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Where it sits in the disease tree

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Part of

↑ Inherited retinal dystrophy (513)

Sub-types of BEST1-related dominant retinopathy

Vitelliform macular dystrophy 2 7 trials

Including sub-types (31) Tagged with BEST1-related dominant retinopathy (1)

Trials to join now! 1

Gene therapy injection aims to halt rare blindness

Disease control Recruiting now

This early-stage trial tests a gene therapy called OPGx-BEST1 for two rare inherited eye diseases that cause vision loss. About 10 adults will receive a single injection into one eye to see if it is safe and to find the best dose. The study will follow participants for 5 years to…

Phase 1/2 • Sponsor: Opus Genetics, Inc • Aim: Disease control

Last updated Jun 27, 2026 12:00 UTC