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.
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