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. 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 […]


  2. COVID-19

    Dear community, This strain of coronavirus is new, which means the knowledge on how it will affect people living with SMA is still limited. However, this is a rapidly changing field so advice will be updated as more is learned. We have built a repository of information, where this information will be uploaded. Leading experts […]


  3. Roche completes recruitment for JEWELFISH clinical trial

    Roche has shared a community update to inform us about recruitment completion for their JEWELFISH clinical trial. This milestone marks the enrolment of 174 participants from across the globe. JEWELFISH is a clinical trial of the small molecule risdiplam. Risdiplam is a small molecule drug taken orally, which targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of […]


  4. Risdiplam shown to improve motor function in people aged 2 to 25 with type 2 or 3 SMA

    Roche’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy First placebo-controlled trial to include adults with SMA demonstrates risdiplam improved or stabilised motor function Medically meaningful and statistically significant results in primary and key secondary endpoints Pivotal SUNFISH Part 2 study population represents broad, […]