EMA New medicine authorised: gene therapy Zolgensma for the treatment of spinal muscular atrophy
EMA has recommended granting a conditional marketing authorisation in the European Union for the gene therapy Zolgensma (onasemnogene abeparvovec) to treat babies and young children with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement. Spinal muscular atrophy is usually diagnosed in the first