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 to file application for Nusinersen to EMA over coming weeks

    Dear members of the SMA community, In response to your requests for information, we want to provide an update on the important progress made in moving the compound development program forward. Today we have achieved a crucial step in the pathway to approval of nusinersen. We have completed the submission of our New Drug Application […]


  2. Roche provides update on splicing modifier RG7916

    Roche is planning to start clinical studies on RG7916 in people with SMA Type 1 and SMA Type 2/3 by the end of 2016 and are awaiting health authority feedback. This feedback will determine final countries, sites and timelines for the studies. An update will be provided in due course. Patients who participated in the […]


  3. Development of LMI070: Novartis provides community update

    Novartis has a product in clinical trial for SMA and wish to share the following update on the development of LMI070. In May, we shared with you news of the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). This decision was made because […]


  4. Nusinersen questions and answers on the latest developments

    In response to a request made by SMA Europe, Biogen has supplied us with a Nusinersen questions and answers (Q&Q) sheet to further explain the significance of the announcement the companies made on 1st August, regarding the development of Nusinersen. Please find the questions and answers below: On August 1, 2016 Biogen and Ionis Pharmaceuticals […]