Gene therapy Zolgensma (onasemnogene abeparvovec) conditionally approved in EU for treatment of spinal muscular atrophy (SMA) in babies and young children up to 21 kilograms

European Commission gave conditional approval to the therapy, whose U.S. price is $2.1 million, for patients with SMA Type 1, the most severe form of the disease, or patients with up to 3 copies of a specific gene that helps doctors predict disease severity.


Reuters Health