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One-off injection will save children from inherited blindness

Mon, 09/09/2019 - 10:18 -- geeta.nair

NHS England is to pay for a cutting-edge genetic treatment that will save children with a rare inherited disorder from going blind.

A one-off injection will prevent babies born with poor sight because of an inherited retinal dystrophies disorder from losing their vision entirely, which usually happens during childhood.

The list price is high, at £613,410 per patient, but the NHS has done a deal with Novartis, the UK supplier of voretigene neparvovec, also known as Luxturna, and the numbers needing treatment are low. The company estimates that 86 people in England could benefit now, and about three to five babies with the genetic mutation are born every year.

Loss of vision can have devastating effects, particularly for children and young people, but this truly life-changing treatment restores the sight of people with this rare and distressing condition,” said NHS chief executive, Simon Stevens, making the announcement at the Health Innovation Expo conference in Manchester.

“For previous generations, curing blindness would literally have been seen as a miracle. Now modern medicine is making that a reality for our patients. Once again the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this revolutionary new form of treatment.

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