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News From the Food and Drug Administration
February 6, 2018

Gene Therapy for Vision Loss

JAMA. 2018;319(5):434. doi:10.1001/jama.2017.21710

A new treatment for children and adults with a rare, inherited form of vision loss is the first gene therapy approved in the United States for a genetic disease.

Voretigene neparvovec-rzyl, marketed as Luxturna, is indicated for patients with confirmed biallelic RPE65 mutation–associated retinal dystrophy. Only patients with mutations on both copies of the RPE65 gene who have sufficient viable retinal cells should receive the therapy. It is a one-time treatment administered by subretinal injection.