BBC News: Gene therapy for rare eye disease set to be offered on NHS

4 September 2019
Source: BBC News

Patients with a rare inherited eye disorder are set to be able to access a new gene therapy BBC news logoon the NHS which slows down sight loss.

Draft NICE guidance recommends the therapy (Luxturna) to treat inherited retinal dystrophies.

The National Institute for Health and Care Excellence estimates just under 90 people in England will be eligible for the treatment.

Drug company Novartis agreed a discount for the NHS on the £613,410 price.
Without the deal, the therapy, voretigene neparvovec, would have breached the regulator’s budget impact test.

‘Breakthrough therapy’

Inherited retinal dystrophies cause the gradual degeneration of the light-sensitive cells on the retina. They are usually diagnosed in childhood.

The condition causes a loss of vision which ultimately leads to almost total blindness. At the moment, there is no treatment.

People with a mutation of the RPE65 gene will be suitable for the new treatment. The gene should provide the instructions to make a protein that is key to normal vision.

Read the full story on the BBC Website

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