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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.
Retina UK is delighted to have awarded a new three-year research grant to Prof Majlinda Lako at Newcastle University for a project entitled: To establish AAV.PRPF31 gene augmentation in PRPF31-deficient RPE and photoreceptor cells and assess its efficacy in restoring RPE and photoreceptor function.
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.
The Retina UK community is sometimes targeted by adverts online or in social media, offering invasive treatments for inherited sight loss.
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.
Joining us for an "Ask the Expert" session is Dr Roly Megaw.
Faye was part of #TeamRetinaUK in 2019. This is what she had to say in the run up to the race:
Whether you're an expert runner, or a rebel *with* a cause, there's no better experience than the London Marathon!
Fundraising for Retina UK means a lot to me - both my dad and my nephew have been affected by inherited sight-loss, and I've seen first-hand the incredible work this charity does.
This October, join Retina UK on Halloween weekend (28 and 29 October 2023) and face your fears by taking on a Halloween skydive at various airfields around the UK.
