News Archive - SMA Europe

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. 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 […]


  2. 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 […]


  3. SMA Europe held its bi-annual meeting in Berlin

    SMA Europe held its bi-annual meeting in Berlin on 13th and 14th October 17, kindly hosted by Initiative SMA, an initiative of the German neuromuscular organisation, Deutsche Gesellschaft für Muskelkranke (DGM). A number of topics were discussed, from the availability and scope of Spinraza™ reimbursement across different European countries, to SMA Europe’s International Scientific Congress for […]


  4. SMA Europe bi-annual meeting – October 2017

    SMA Europe is hodling its second annual meeting of the year, kindly hosted by the German neuromuscular disease association, DGM (Deutsche Gesellschaft  für Muskelkranke e.V.) in Berlin between 13th & 14th October 2017. The programme will include: Individual meetings with the main pharmaceutical companies involved in the development of therapies for SMA (Avexis, Biogen, Novartis […]


  5. AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1

    The U.S. Food and Drug Administration (FDA) has notified AveXis that, based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with SMA Type 1 using the intravenous (IV) formulation produced by the company’s Good Manufacturing Practice (GMP) commercial manufacturing process. The company plans […]