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. Zolgensma Summary of Product Characteristics Updated

    The Summary of Product Characteristics (SmPC) for Zolgensma has been updated. The SmPC is a document describing the properties and the officially approved conditions of use of a medicine. Summaries of product characteristics form the basis of information for healthcare professionals on how to use the medicine safely and effectively. Novartis Gene Therapies is committed […]


  2. SMART, a phase 3b clinical trial of Zolgensma, to be initiated soon

    Novartis Gene Therapies will initiate SMART, a Phase 3b clinical study to further evaluate the safety and efficacy of Zolgensma® (onasemnogene abeparvovec) in people living with SMA, weighing between 8.5 kg and 21 kg, following a single intravenous (IV) infusion. Clinical data from this study will supplement emerging real-world evidence and use of onasemnogene abeparvovec. […]


  3. 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 […]