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. Spinraza (Nusinersen) approved by the FDA

    Spinraza (Nusinersen)Yesterday, the FDA announced that it has approved Spinraza (Nusinersen) to treat spinal muscular atrophy, making it the first-ever FDA-approved therapy for SMA, a product which addresses the underlying genetic cause of the condition by making the back-up gene, SMN2, produce more functional SMN protein. This milestone has been achieved as a result of the cooperation between all stakeholders: researchers, families & groups, companies and regulators, all working together as one community.

    The approval from the FDA for all SMA (paediatric and adult), is the broadest possible label and has happened as a result of Biogen’s rigorous clinical development plan for Spinraza (Nusinersen).

    Here in Europe, we eagerly await the decision from the European Medicines’ Agency (EMA).

    Please read Biogen’s press release!