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Leber Congenital Amaurosis

Prevalence: ~100K

ophthalmologic

Official·OMIM:204000 Official·ORPHA65

Inherited retinal dystrophy causing severe vision loss from birth.

Approved Therapies

Luxturna (voretigene neparvovec)

FDA Approved12/19/2017AAV Gene Therapy

Associated Genes

RPE65Retinoid Isomerohydrolase RPE65

Chromosome 1Autosomal Recessive