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. 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. […]


  2. 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 […]


  3. EMA recommends approval of risdiplam for 5q SMA

    We are delighted to share with you that the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), adopted a positive opinion recommending the approval of risdiplam for the treatment of 5q SMA, in people 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type […]