The Medical Letter on Drugs and Therapeutics
Voretigene Neparvovec-rzyl (Luxturna) for Inherited Retinal Dystrophy
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The FDA has approved voretigene neparvovec-rzyl intraocular suspension (Luxturna – Spark), an adeno-associated virus vector-based gene therapy, for treatment of confirmed biallelic RPE65 mutation-associated retinal dystrophy in patients who have viable retinal cells. It is the first gene replacement therapy approved in the US that targets a disease caused by mutations in a specific gene and the first pharmacologic treatment to be approved for this disorder.

THE DISEASE — Inherited retinal dystrophies are primarily caused by gene defects in photoreceptors and retinal pigment epithelial (RPE) cells; more than 200 different genes have been identified. Biallelic RPE65 mutation-associated retinal dystrophy is an autosomal recessive disorder affecting about 1000-3000 persons in the US. The RPE65 gene encodes the enzyme all-trans-retinyl ester isomerase, which is crucial in the ... more      

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Title: Voretigene Neparvovec-rzyl (Luxturna) for Inherited Retinal Dystrophy
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