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. 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, […]


  2. 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 […]


  3. Roche provides an update on the clinical development of risdiplam

    Summary 12-month data from the pivotal SUNFISH Part 2 study submitted to the FDA in February, leading to a 3 month review extension of the New Drug Application (NDA) On track to submit a Marketing Authorisation Application (MAA) to the European Medicines Agency in mid-2020 Regulatory filings have been submitted in Brazil, Chile, Indonesia, Russia, […]


  4. European Medicines Agency grants positive recommendation for conditional marketing authorisation of gene therapy product onasemnogene abeparvovec (Zolgensma®)

    On 27th March, the European Medicines Agency recommended a conditional approval of the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy. SMA Europe is thrilled that a second therapy that addresses the causes of this life-limiting disease has moved closer to patients. It is a culmination of efforts that have been […]