ProQR clinical trial for Leber’s Congenital Amourosis (LCA) 10
Biopharmaceutical company ProQR is conducting a Phase 2/3 clinical trial (Illuminate) to measure the safety and effectiveness of RNA therapy, Sepofarsen which aims to treat Leber’s Congenital Amourosis (LCA) …
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We are here for you
We are all living in challenging times and this is causing uncertainty and anxiety for many in our community. In March we recorded a significant increase in demand for our …
Ask the expert
Would you like to speak with an expert who cares for lots of patients with inherited sight loss conditions and has a detailed knowledge of the field? Consultant Ophthalmologist Dr …
Positive early findings from ProQR trial of USH2A therapy
Biotechnology company ProQR has announced encouraging results from its early analysis of the phase 1/2 trial of QR-421a, an innovative approach to treating sight loss caused by mutations in a …
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Useful links during Covid-19 pandemic
The following list of useful links have been compiled by the team at Retina UK to provide information and support for our community. The list is updated regularly. However, in …
Retina UK COVID-19 response
During these uncertain times, the health and wellbeing of our community is our upmost priority at Retina UK. In light of Government guidance we have made the difficult decision to …
First clinical trial participant receives CRISPR-based therapy
An American living with Leber congenital amaurosis 10 (LCA10) has become the first clinical trial participant in the world to receive a CRISPR gene editing treatment in vivo (inside the …
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Popular historic drama features retinitis pigmentosa
We were delighted to support the BBC1 drama Call the Midwife production team with their historical research for an episode featuring a character living with retinitis pigmentosa (RP). Professor John …
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First patient receives ground-breaking treatment
In January 23-year-old Jake Ternent became the first person with an inherited sight loss condition to be treated in the UK with Luxturna (voretigene neparvovec) for Leber congenital amaurosis (LCA). …
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Gene therapy shows promise in tackling X-linked RP
Early results from clinical testing of a gene therapy to treat X-linked retinitis pigmentosa (XLRP) have shown partial reversal of sight loss in some patients. The treatment targets retinal degeneration …
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Genetic testing: an essential topic for discussion with the IRD communtiy
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 …
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Latest update from ReNeuron stem cell trial
ReNeuron has announced an encouraging update on the progress of its Phase 1/2 trial of a stem cell-based therapy for retinitis pigmentosa, describing the most recent results as a “clear …
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