Autosomal dominant inheritance
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.
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Phase 3 clinical trial has been given approval by FDA
The FDA has given Ocugen permission to start their phase 3 gene therapy clinical trial for retinitis pigmentosa (RP).
Light sensing small molecule given green light
Biotechnology company Kiora Pharmaceuticals has been given the go-ahead to start in-human testing of a novel “small molecule” treatment, known as KIO-301, for advanced vision loss in retinitis pigmentosa (RP). The phase 1b clinical trial will take place in Adelaide, Australia, and will begin enrolling participants towards the end of 2022.
What we do
Our vision is a world where everyone with inherited sight loss is able to live a fulfilling life.
Meira GTx announces positive data from phase clinical trial
Biotechnology company MeiraGTx has announced encouraging news from its phase 1/2 clinical trial of botaretigene sparoparvovec (previously known as AAV-RPGR), a gene therapy aimed at X-linked retinitis pigmentosa caused by faults in the RPGR gene.
Looking after your vision
It is important to avoid harming your vision, particularly if you have an inherited sight loss condition.
Getting a referral
If you have a diagnosis of an inherited retinal condition, or you are a close relative of somebody living with one of these conditions, you are entitled to access testing and genetic counselling.
Research news round-up – Spring 2024
A round-up of the latest research into inherited retinal conditions - February 2024.
Beware of online adverts about treatments
The Retina UK community is sometimes targeted by adverts online or in social media, offering invasive treatments for inherited sight loss.