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. Biogen provides information on upcoming Spinraza EU label

    In response to our request, Biogen has provided us with information and addressed our questions and concerns regarding the upcoming SPINRAZA (nusinersen) EU label (Prescribing information and Package Leaflet) update that will include information on the potential risk of hydrocephalus. All medicines are evaluated for the benefit they provide to patients as well as the […]


  2. 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 […]


  3. Novel protective gene for SMA opens new therapeutic options

    The research group of Prof. Dr. Brunhilde Wirth, Head of the Institute of Human Genetics at the University Hospital Cologne and Chair of our Scientific Advisory Board, succeeded to identify calcineurin-like EF-hand Protein 1 (CHP1) reduction as a novel protective modifier for SMA. The results of this study have been published in Brain, an international highly renowned journal. […]


  4. Our 2018 – 2020 activities and initiatives

    In April this year, a new 2-year strategy was set and a new working structure established. Our new structure   Elections Presidency: Mencía de Lemus from our Spanish organisation, FundAME vice-Presidency: Nicole Gusset from our Swiss organisation, SMA Schweiz Treasury: Inge Schwersenz from our German organisation, DGM     Executive Committee As SMA Europe continues to grow, it […]


  5. An animation to explain lumbar punctures

    A lumbar puncture, also called spinal tap, is a medical test. It may be applied to children and young people in order to diagnose diseases of the nervous system or to give a medication (such as Spinraza™). The department of Paediatric Neurology and Muscle Diseases at Freiburg University Medical Centre has produced this animation to […]