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Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Evidence-based recommendations on idebenone (Raxone) for treating visual impairment in Leber’s hereditary optic neuropathy in people 12 years and over.
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