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®, the first gene therapy to treat SMA, receives conditional marketing authorisation in the European Union

    On 19th May, the European Commission issued a conditional marketing authorisation for the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy. This is the second drug developed to treat this severe neuromuscular disorder to receive approval in the European Union. Onasemnogene abeparvovec, marketed as Zolgensma®, is authorised for use in treating […]


  2. Scholar Rock reports delays in interim trial results due to COVID-19

    Scholar Rock reported on the recent progress and upcoming milestones for the TOPAZ Phase 2 clinical trial of SRK-015 in patients with Type 2 or 3 SMA, in the light of the COVID-19 pandemic. SRK-015 is a muscle-directed therapy that aims to reverse or restrict the muscle atrophy and weakness experienced by SMA patients. The drug is […]


  3. DEVOTE Trial – First patient treated

    Based on our request for an update, Biogen has provided us with the following information on its global clinical study DEVOTE. The study is designed to evaluate the safety, tolerability and potentially improved efficacy of nusinersen when administered at a higher dose than currently approved for the treatment of SMA. The Phase 2/3 randomised, controlled, […]


  4. Roche COVID-19 Response Measures

    Summary This news item contains information relating to: The supply maintenance for risdiplam Roche’s ongoing clinical trials & support services for trial participants An update to Pre-Approval Access / Compassionate Use (PAA/CU) Programme for risdiplam Over the past few weeks, Roche, alongside its partners, has been monitoring the COVID-19 global pandemic to better understand the […]