First patient undergoes Luxturna gene therapy on NHS – PharmaTimes

The NHS has reported treating its first patient with Novartis Luxturna (voretigene neparvovec) a revolutionary new gene therapy that can restore eyesight, as part of its NHS Long Term Plan.

The therapy is for those born with an inherited retinal disorder - Lebers Congenital Amaurosis (LCA) - who have poor sight which swiftly deteriorates, with many ultimately losing their vision completely in childhood.

The life-changing treatment for children and adults is the first in a new generation of gene therapies that can be directly administered to patients, in this case through an injection. Many patients in the trials have recovered their night time vision with the treatment.

Jake Ternet, patient at Moorfields Eye Hospital was the first in the UK to receive the treatment.

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.

Once again, the NHS is at the forefront of the genomic revolution with patients in England among the first to benefit from this new form of treatment a modern day miracle as part of the Long Term Plan.

Back in September last year, The National Institute for Health and Care Excellence (NICE) recommended the use of Luxturna on the NHS for certain patients with RPE65-mediated inherited retinal dystrophies in those with vision loss.

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First patient undergoes Luxturna gene therapy on NHS - PharmaTimes

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