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PRPF31-related retinopathy

MONDO:0800395

An inherited retinopathy caused by variants in the PRPF31 gene.

29 clinical trials for this condition and its sub-types, 1 tagged with PRPF31-related 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 PRPF31-related retinopathy

Retinitis pigmentosa 11 4 trials

Including sub-types (29) Tagged with PRPF31-related retinopathy (1)

New eye injection shows promise for rare Blindness-Causing gene mutation

Disease control Completed

This early-stage study tested a new drug, VP-001, given as an injection into the eye to see if it is safe for people with a rare genetic eye disease caused by a PRPF31 mutation. Seventeen adults took part. The main goal was to check for side effects, not to measure vision improve…

Phase 1 • Sponsor: PYC Therapeutics • Aim: Disease control

Last updated Jun 27, 2026 13:00 UTC