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. A Biogen community update on Spinraza NURTURE study

    Based on our request for an update, Biogen has provided us with the following information about new results from their NURTURE study, a study of pre-symptomatic patients with SMA, recently presented at the virtual Cure SMA Research & Clinical Care Meeting, which took place from 10th to 12th June 2020. NURTURE is an ongoing, Phase […]


  2. 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 […]


  3. 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 […]