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. FDA grants priority review to risdiplam for the treatment of SMA

    The United States’ Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for risdiplam (RG7916) for the treatment of SMA. The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA. […]


  2. SMA Europe awards 4 new grants to cutting-edge research projects

    SMA research receives vital funds from SMA Europe. Based on the recommendations received by its Scientific Advisory Board, SMA Europe awarded a total of 544,900 Euros to 4 cutting-edge SMA research projects through its 10th Call for Research Proposals. The Scientific Advisory Board reviewed and scored 19 very high level SMA research proposals. As only […]


  3. FDA puts partial hold on AVXS-101 intrathecal clinical trials for SMA

    Novartis announced this morning that the Food and Drug Administration (FDA) in the US has placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study. Zolgensma® , also known as AVXS-101 intravenous administration is not impacted and remains available in the US. Novartis is […]