ProQR makes progress with potential treatment for LCA
Despite the challenges of the pandemic, researchers and pharmaceutical companies are still making progress towards delivering new treatments for inherited sight loss.
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Despite the challenges of the pandemic, researchers and pharmaceutical companies are still making progress towards delivering new treatments for inherited sight loss.
Sepul Bio begins clinical trial of ultevursen for USH2A-associated RP
Inherited retinal dystrophies (IRDs) are the leading cause of blindness in working-age people in the UK, and children as young as eighteen-months are regularly diagnosed.
There are many ongoing clinical and laboratory studies around the world, exploring innovative approaches to treating inherited sight loss.
We are proud to work collaboratively with a number of corporate partners to enable our community to live fulfilled lives today as well as supporting the pharmaceutical industry in their mission to develop potential new treatments.
In recent years there has, understandably, been a lot of excitement around cutting-edge therapies that target the specific genetic faults underlying inherited sight loss.
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Catherine L’Estrange, aged 11 from London has become the first individual in the UK to receive an investigative gene therapy treatment for retinal dystrophy associated with the BBS10 gene.
Inherited progressive sight loss is caused by a range of rare genetic conditions. We specialise in those which affect the retina.
Biotechnology company ProQR has announced encouraging results from its phase 1/2 clinical trial of an innovative treatment for sight loss caused by faults in a specific section of the USH2A gene.