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ProQR has published the top-line results from its Phase 2/3 “Illuminate” trial of the RNA therapy sepofarsen for Leber congenital amaurosis type 10 caused by a specific mutation in the CEP290 gene.
Autosomal dominant inheritance means that the faulty gene is on an autosome (any chromosome other than the sex chromosomes) and that only one faulty copy is needed to cause sight loss.
A round-up of the latest research into inherited retinal conditions - February 2024.
UK researchers have discovered that passing a weak electrical current between electrodes on a person’s scalp may lead to a reduction in frequency of the visual hallucinations experienced by some people living with sight loss.
Disulfiram (Antabuse), FDA-approved for deterring alcohol, is in a phase 1 trial at the University of Washington to improve vision in retinitis pigmentosa.
The latest research news from Retina UK.
SpliceBio has begun the dose-expansion portion of their phase 1/2 clinical trial of SB-007, a dual adeno-associated viral vector gene therapy for Stargardt disease.
Ocugen’s gene therapy candidate, OCU410ST, is an experimental treatment designed to slow vision loss in Stargardt disease, an inherited retinal disease (IRD) with no approved therapies currently.
Inside: Our intrepid Sahara Trek #TeamRetinaUK tell us about their incredible experience. 2026 is now fully underway and we are excited to tell you about our plans. Could you take part in a challenge event or volunteer to do a bucket collection in London for Retina UK?
A recently published study has described numerous disease mechanisms that appear to be common across different types of inherited sight loss, suggesting that there is significant potential for drug treatments that could work regardless of the underlying causative gene.
