News | 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. Scholar Rock announces results from TOPAZ phase 2 trial in Type 2 and 3 SMA

    What is apitegromab? Apitegromab (SRK-015) is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA. The drug is an antibody that very specifically targets an important protein called myostatin, which restricts muscle mass and strength. Scholar Rock believes that by preventing the activation of […]



    Come and join our dedicated team! To lead daily activities to ensure sustainability and adequate organisational process, SMA Europe, a non-for-profit umbrella organisation of European 23 national spinal muscular atrophy (SMA) patient and research associations across 22 European countries, is seeking an experienced Operations Manager. With unique insight and expertise, SMA Europe brings effective treatments […]


  3. European Commission approves Evrysdi for the treatment of 5qSMA

    Following our request for regular updates, Roche is delighted to inform us that today the European Commission (EC) approved Evrysdi™ (risdiplam), the first and only at home treatment for SMA, with proven efficacy in adults, children and babies two months and older. Risdiplam was reviewed under the European Medicine Agency’s (EMA) accelerated assessment programme, intended […]


  4. Alliance strives to bridge the newborn screening gap to include SMA!

    It’s time for Europe to bridge the newborn screening gap and include spinal muscular atrophy!   The European Alliance for Newborn Screening in Spinal Muscular Atrophy calls upon the government of Europe to include a test for spinal muscular atrophy in national newborn screening programmes.   Worldwide newborn screening projects and programmes for SMA clearly […]