Posted by Michael Wonder on 04 Sep 2019
NICE recommends novel gene therapy treatment for rare inherited eye disorder
4 September 2019 - It's estimated that 86 people would be eligible for treatment with voretigene neparvovec in England.
A novel gene therapy treatment for rare inherited eye disorder has been recommended by NICE for use on the NHS.
The draft highly specialised technologies guidance recommends voretigene neparvovec (also called Luxturna and made by Novartis Pharmaceuticals UK) for people with vision loss caused by inherited retinal dystrophy from confirmed RPE65 gene mutations and who have enough viable retinal cells.
