AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA)
Zolgensma® (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene Zolgensma has de…