Research update – ProQR
In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
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In February 2022, ProQR announced a disappointing outcome for their phase 2/3 trial of sepofarsen for Leber congenital amaurosis type 10 (LCA10).
James Birtley is a Retina UK supporter and volunteer who is living with retinitis pigmentosa. He is also a scientist with a particular interest in the structure of proteins, the complex molecules that form the building blocks of our bodies.
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.
It is important to avoid harming your vision, particularly if you have an inherited sight loss condition.
You may have heard that a cell-based treatment approach (sometimes referred to as a “stem cell treatment”), developed by a company called ReNeuron, is being tested in a clinical trial at Oxford Eye Hospital and other centres in the US and Europe.
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.
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.
Inflammation and retinal degeneration in retinitis pigmentosa: Can lessons learned in the lab help us identify treatments in the clinic?
We talk to BBC reporter Ramadan Younes and Professor Robert Maclaren following the BBC documentary Blind Faith