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Ten children regain sight with gene therapy – breaking latest news

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Ten children have regained their sight at the Vanvitelli University hospital in Naples thanks to a gene therapy, the voretigene neparvovec from Novartis, for hereditary retinal dystrophies. It is the first place in Italy where this therapy is applied that deals with a rare form of hereditary retinal dystrophy, linked to mutations in both copies of the RPE65 gene and which has been tested for about 15 years by Vanvitelli with the Telethon Foundation and the Children’s Hospital of Philadelphia. The children came from Piedmont, Lombardy, from many other Regions, Naples thus becomes a point of reference at the national level for the treatment of rare diseases of the retina. “The ten patients treated – 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 have a profound scientific and clinical value as well as to testify that, in a degenerative pathology, the way of early treatment is the winning one “.

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