Rare diseases: Rondena (Novartis), 'Gene therapy addresses the cause of SMA and blocks its progression'

“Novartis started with an important unmet medical need and developed an answer for children born with SMA, a rare genetic disease. Gene therapy can address the cause of the disease, providing replacement for a missing or non-functioning gene and, therefore, blocking the progression of the disease.” Thus Roberta Rondena, Country value & access head of Novartis, on the sidelines of the press conference with which the pharmaceutical company announced the green light from Aifa to the extension of the reimbursement of onasemnogene abeparvovec, the first gene therapy approved in Italy for the treatment of spinal muscular atrophy (Sma) type 1, also to children with spinal muscular atrophy type 2 (Sma2).