Gene therapy restores 10 kids' sight

Innovative treatment at Naples' Vanvitelli Hospital

(ANSA) - ROME, OCT 18 - Ten children from various regions of central and northern Italy have regained their eyesight thanks to the first gene therapy, Novartis's voretigene neparvovec, for hereditary retinal dystrophy, carried out at Naples' Ateneo Vanvitelli Hospital, sources said Monday.
    The therapy, approved and reimbursed in Italy, tackles a rare form of hereditary retinal dystrophy, linked to mutations in both copies of the RPE65 gene, and the therapy began around 15 years ago, with phase 1 testing via collaboration between Vanvitelli University, the Telethon Foundation and the Children's Hospital in Philadelphia. (ANSA).


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