Search results
Opus Genetics has launched an early-stage clinical trial of OPGx-MERTK, an investigational gene therapy for MERTK-related Retinitis Pigmentosa (RP), a condition that causes progressive vision loss due to photoreceptor degeneration.
Are you affected by inherited sight loss? We are here to help.
X-linked inheritance means that the faulty gene is located on the X chromosome, the larger of the two sex chromosomes.
Biotechnology company ProQR has announced that its clinical trials of sepofarsen for Leber congenital amaurosis type 10 and ultevursen for USH2A-mediated retinitis pigmentosa will be wound down with immediate effect.
Prof Mariya Moosajee at Moorfields Eye Hospital has asked us to share the message below about the clinical trial she is running. This trial is for a treatment that targets a particular section of the USH2A gene.
As Luxturna reaches the clinic and other gene-specific therapies for inherited retinal disease get closer to the end of the development pipeline, it is becoming ever more important that affected families can access a genetic diagnosis, potentially opening up choices around treatment and clinical trial participation.
Disulfiram (Antabuse), FDA-approved for deterring alcohol, is in a phase 1 trial at the University of Washington to improve vision in retinitis pigmentosa.
Are you a parent or carer of a child or young person living with an inherited retinal dystrophy?
We have secured some in-demand slots for bucket collections on the London Underground. Can you spare a few hours to support us in April, during our 50th anniversary year and raise awareness to thousands of people?
