NHS go ahead for £600,000 gene therapy to treat rare eye disorder

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A new gene therapy has been used to treat patients with a rare inherited eye disorder which causes blindness, reports BBC News. It’s hoped the National Health Service (NHS) treatment will halt sight loss and even improve vision.

The treatment costs around £600,000 but NHS England has agreed a discounted price with the manufacturer Novartis.

The report says Luxturna (voretigene neparvovec), has been approved by The National Institute for Health and Care Excellence (NICE), which estimates that just under 90 people in England will be eligible for the treatment.

The gene therapy is for patients who have retinal dystrophy as a result of inheriting a faulty copy of the RPE65 gene from both parents. The gene is important for providing the pigment that light sensitive cells need to absorb light. Initially this affects night vision but eventually, as the cells die, it can lead to complete blindness.

Professor Stephen Powis, NHS medical director, said: “Loss of vision can have a devastating effect, particularly for children and young people, but this truly life-changing treatment offers hope to people with this rare and distressing condition.”

Full BBC News report

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