Blind babies to have eyesight restored by revolutionary £613,000 gene therapy available on the NHS – The Sun

BLIND children will have their eyesight restored by a revolutionary new gene therapy on the health service.

The life-changing treatment — usually £613,410 a patient but supplied at an undisclosed, reduced price — will be available for everyone from 2020, the NHS says.

Boss Simon Stevens told a conference in Manchester that curing blindness was once seen as a miracle but added: “Now modern medicine is making that a reality for our patients.”

Babies born with inherited retinal dystrophies disorder have poor sight which swiftly deteriorates. Most lose their vision completely in childhood.

The condition blocks messages for making proteins in the eye that are essential for normal vision. But the therapy — voretigene neparvovec — recreates those processes.

It is the first in a new generation of gene therapies that can be directly administered to patients — such as via injection.

Mr Stevens added: “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.”



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He also praised manufacturer Novartis for helping to make the to treatment, also known as Luxturna, affordable.

Eye expert Professor Robert MacLaren said: "The progression of inherited retinal degeneration caused by RPE65 gene mutations leads to blindness, which has a profound effect on the lives of affected patients and their carers."

He hailed the therapy's "potential to improve vision in children and adults, and enabling them to participate fully at school, work and in their private lives”.

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