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. EMA recommends approval of risdiplam for 5q SMA

    We are delighted to share with you that the Committee for Medicinal Products for Human Use (CHMP), of the European Medicines Agency (EMA), adopted a positive opinion recommending the approval of risdiplam for the treatment of 5q SMA, in people 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type […]


  2. Roche provides the community with a summary of their 2020 work

    Roche has provided us with a summary of its work in the community throughout this year, including information about the risdiplam clinical development programme and progress towards their goal of making risdiplam available to all patients who can benefit from the treatment. Ensuring patient safety, uninterrupted access to treatment and to support the wider community: […]


  3. Update on the clinical development of risdiplam

    Following our request to receive important and timely information about the risdiplam clinical development programme, Roche has shared with us an update on part 1 of the FIREFISH clinical trial. FIREFISH is an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the […]