Rare diseases, Pallara (Sma Families): “One-shot administration of gene therapy is a revolution”

“Having the possibility of receiving a therapy that has a 'one shot' administration, i.e. only once in a lifetime, making you free even from repeated hospital admissions, is a huge revolution and responds to the ever-present and reiterated need of our community to receive therapies as effective and safe as possible and which can lead to a radical change in the course of the pathology”. Anita Pallara, president of Famiglie Sma, said this on the sidelines of the press conference organized by Novartis in Rome, during which the green light from Aifa was announced for the expansion of the reimbursement criteria for onasemnogene abeparvovec, the first gene therapy approved in Italy for the treatment of spinal muscular atrophy (Sma), already available for Sma type 1 and from today also for children with type 2 (Sma2).