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 releases statement on CHERISH data

    At our request, Biogen sent us a community update on the end of study results from CHERISH, the Phase 3 trial evaluating nusinersen for the treatment of individuals with later-onset SMA, which have now been published in the New England Journal of Medicine (NEJM). The full manuscript entitled, “Nusinersen Versus Sham Control in Later-Onset Spinal […]


  2. AveXis provides community update on plans for AVXS-101 in Europe

    AveXis has provided SMA Europe with a community update on plans for AVXS-101 in Europe. AVXS-101 is a gene therapy product for SMA. STR1VE EU OVERVIEW: STR1VE EU is expected to start enrolling patients in the first half of 2018 in patients with SMA Type 1, studied at multiple centres across the European Union. ADMINISTRATION: […]


  3. AVEXIS announced Phase 1 clinical trial of AVXS-101 for SMA Type 2

    Avexis has been granted authorisation to start Phase 1 clinical trial of AVXS-101 for patients with SMA Type 2 by the U.S. Food and Drug Administration (FDA), based on review of data submitted. The company plans to initiate this trial immediately. The open-label, dose-comparison, multi-center Phase 1 trial named STRONG, is designed to evaluate the […]


  4. The patient voice at EMA

    The European Medicines Agency (EMA) is the European Union agency responsible for the evaluation and supervision of medicines. Hear the voices of EMA’s stakeholders – Patients and healthcare professionals representatives explain the value of working with the Agency for better decision-making and how important it is to ensure your voice is added to the regulatory […]


  5. Roche provides community statement regarding SUNFISH

    Roche provides community statement regarding SUNFISH, a clinical trial of RG7916 for SMA Types 2/3. An interim analysis was recently presented at the International Conference of the World Muscle Society in France. In this analysis, all participants (51 people) had received RG7916 for 28 days or longer. SMN protein increased by up to two and […]