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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.
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“All the promising research makes you feel like there is light at the end of that very dark tunnel after all.”
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
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).
Next month, Anisha and Sheena will take on The Thames Path Challenge. Together, they explain why they decided to join #TeamRetinaUK.
In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
Early 2020 marked an important milestone for the Retina UK community, when the first person with an inherited retinal condition received NHS treatment to potentially slow or even stop the progression of their sight loss.
TPT and Sight Loss Councils: Cut it back campaign
In 2014 we funded an exciting new collaborative project. The UK Inherited Retinal Dystrophy Consortium (UKIRDC) Project brought together the four largest research groups in the UK specialising in inherited retinal diseases (IRDs).
