Emerging treatments for Retinitis Pigmentosa

Abstract

Retinitis Pigmentosa (RP) is a class of rare-inherited retinal dystrophies which can lead to blindness and is characterised by pigment deposits predominantly present in the periphery of the retina. The hereditary pattern of RP follows a Mendelian inheritance pattern. The onset of RP typically occurs during the early teenage years with difficulties in dark adaptation, concurrent nyctalopia and reduced visual field (VF) which leads to tunnel vision and gradual reduction in central vision with a risk of blindness. Currently, no standard treatment for RP is available. One medicinal product (MP), Luxturna, which consists of voretigene neparvovec, a gene transfer vector, is authorised in the European Union (EU) and United States (US) to treat patients with inherited retinal dystrophies, caused by biallelic RPE65 mutations, and who have sufficient viable retinal cells.