The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Novartis announces FDA filing acceptance and Priority Review of AVXS-101

    Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA®(onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1, a […]


  2. Roche provides community update on FIREFISH & SUNFISH studies of risdiplam

    Roche has provided us with the following statement on the FIREFISH and SUNFISH studies of risdiplam: Dear SMA community, We are happy to share an update on our FIREFISH and SUNFISH studies of risdiplam. We would also like to acknowledge the tremendous support and partnership of SMA Patient Groups around the world and to thank […]


  3. RG7916 community update from Roche

    Roche has shared with us an update of the RG7916, now called risdiplam, programme, which has reached a number of milestones. Risdiplam is an SMN2 splicing modifier which is given daily by mouth or by g-tube as a liquid and distributes widely throughout the body. Risdiplam has been designed to help the SMN2 gene produce more […]