Rare diseases, Pane (Cattolica): “Pre-symptomatic treatment allows a child to live a normal life”

The extension of the reimbursement of gene therapy to children with Sma2, as well as with Sma1, “represents a real revolution, because it allows us to treat even children who would not have walked without this treatment. If we manage to treat them very early, when the signs of the disease have not yet arrived, we allow them to live a normal life.” These are the words of Marika Pane, director of the Nemo Pediatric Clinical Center of the Gemelli Polyclinic in Rome and associate professor in Child Neuropsychiatry at the Catholic University, commenting on the extension of the reimbursement of onasemnogene abeparvovec, the first gene therapy approved in Italy for the treatment of spinal muscular atrophy (Sma), including children with spinal muscular atrophy type 2 (Sma2), as well as those with Sma1. Aifa's green light to the expansion of the reimbursement of the treatment was at the center of the press conference organized by Novartis in Rome.