First NHS patients receive new treatment for Leber’s congenital amaurosis

Patients living with Leber’s congenital amaurosis (LCA) have started to receive a new gene therapy on the NHS that has the potential to restore sight.

The treatment, voretigene neparvovec, is directly administered through an injection, with many patients in trials recovering their night-time vision.

There has previously been no available treatment for LCA in the UK and it is estimated that 100 patients could benefit from the new gene therapy.

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