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. Spinraza improves motor function in children with later-onset SMA

    Biogen presented data from the end of the Phase 3 study on Spinraza™ (CHERISH) at a plenary session of the American Academy of Neurology (AAN) annual meeting in Boston on 24th April 2017. This data demonstrated a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset (most likely to develop […]


  2. Biogen EAP Update – 13 April 2017

    Dear members of the SMA community, In response to your request for information, we would like to provide you with an update on Biogen’s global Expanded Access Program (EAP). Based on the high unmet need in SMA, and to bridge the gap between the closure of the shamcontrolled phase 3 ENDEAR study and regulatory approval […]


  3. Roche has provided an update on Olesoxime and RG7916

    Roche has provided an update on the development of neuroprotective agent Olesoxime and small molecule RG7916, which acts on the SMN2 gene. This updates includes information on which trials are currently recruiting, what the eligibility criteria are and how to participate in a trial as well as information on trial design. You can also find […]


  4. New phase 3 ENDEAR data presented by Biogen

    New phase 3 ENDEAR data presented by Biogen at the British Paediatric Neurology Association (BPNA) annual conference in Cambridge last week, show that SPINRAZA™ (nusinersen) significantly reduces the risk of death or permanent ventilation in SPINRAZA-treated infants with SMA compared to untreated infants. In August 2016, Biogen reported that ENDEAR met its pre-specified primary endpoint […]