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 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 of enlargement of the visual field, increase of vision at near, far and in low light conditions, have a profound scientific and clinical value as well as testifying that, in a degenerative pathology, the path of early treatment is the winning one “.
A success also celebrated by the governor of Campania, Vincenzo De Luca who also saw regional contributions: “Being the first reality in Europe – he said – on the ophthalmology clinic aimed at the pediatric age must fill us with pride and great satisfaction, I imagine first of all for the team but for all of us because this is truly an extraordinary result. Among the patients treated at Vanvitelli there was no Campania, they came from Piedmont and Lombardy, Regions presented as the excellence of our country: we are drastically reducing the flows of passive mobility and we will expand the communication and information on the things we have. in Campania, the more these flows are destined to shrink to zero. Families who stayed a month in Naples to follow the children will no longer be forced to go to Paris or Germany and we are proud of this work, thanks to the professionals, researchers, clinicians and surgeons engaged in this truly extraordinary activity ».
Success is the result of years of research: «An extraordinary result – affirms the rector of the Vanvitelli University, Gianfranco Nicoletti -. Ten visually impaired children have regained their sight, I have to thank the Campania Region which has invested in gene therapy and our Ophthalmology for the excellent work that places us among the first centers in Europe for treatment in pediatric age ». The treatment, underlines Simonelli, highlights that «our Center in Europe has the largest number of pediatric patients treated with Luxturna. Until the beginning of the year we had no approved therapies for the treatment of hereditary retinal dystrophies, while now we have a one-off therapy available that allows us to offer real hope to patients “.
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