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. 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 […]


  4. Novartis releases update on the branaplam clinical trial

    Novartis has released the following update on behalf of the branaplam program team: Dear SMA community, For years, our researchers have been working hard to develop a new treatment for SMA. One promising compound in our neuroscience pipeline is LMI070, which started development for the treatment of SMA a few years back. In 2016, we […]