Gene therapy restores sight to ten children

(ANSA) - NAPLES, 18 OCT - Ten children, coming from different regions of central and northern Italy, have regained sight thanks to the first gene therapy, voretigene neparvovecdi Novartis, for hereditary retinal dystrophies carried out at the Vanvitelli University hospital in Naples. Therapy, approved and reimbursed in Italy, faces a rare form of hereditary retinal dystrophy, linked to mutations in both copies of the RPE65 gene, which saw its onset, about 15 years ago, with a phase I trial carried out with the collaboration between the Vanvitelli University, the Telethon Foundation and Philadelphia Children's Hospital.

    The success of the treatments makes Naples a national point of reference for the treatment of rare diseases of the Arezzo area and, underlines the university, shows the effectiveness of the collaboration between public and private more innovative therapies. "The ten treated patients - explains Francesca Simonelli, Full Professor of Ophthalmology and Director of the Ophthalmology Clinic of the University of Campania Luigi Vanvitelli - can now write, read and move independently. The results we have obtained include enlargement of the visual field, increase in visual capacity up close, from afar and in low light conditions have a profound scientific and clinical value as well as testifying that, in a degenerative pathology,the way of early treatment is the winning one.

   Furthermore, in 2019, authorized by AIFA, we treated the first two children in Italy, and today, almost 2 years after the therapy, we can confirm an absolute stability of the results and a good safety profile, data that make us confident that what we have achieved in terms of visual ability will last. in the long term ". (ANSA).