Therapy development updates
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
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Tyne & Wear Local Peer Support Group
Saturday 28 June, 1.00pm - 7.00pm - In-person - Come and join Colin Hetherington and the group for their latest social event at the Tyneside Irish Centre in Newcastle.
Antabuse moves into clinical trial for people with RP
Disulfiram (Antabuse), FDA-approved for deterring alcohol, is in a phase 1 trial at the University of Washington to improve vision in retinitis pigmentosa.
Information and support
Are you affected by inherited sight loss? We are here to help.
Genetic testing: an essential topic for discussion with the IRD community
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.
ProQR announces suspension of LCA10 and USH2A clinical trials
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.
x-linked inheritance
X-linked inheritance means that the faulty gene is located on the X chromosome, the larger of the two sex chromosomes.
Parents and Carer’s Peer Support Group
Are you a parent or carer of a child or young person living with an inherited retinal dystrophy?
Sirius clinical trial – Exon 13 mutation of USH2A
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.