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. Roche ends development of olesoxime

    It was announced today that Roche has taken the difficult decision of ending the development of neuro-protective compound Olesoxime. In 2016 Roche started the OLEOS study, an open label extension study evaluating the long term safety and effectiveness of olesoxime. Many discussions with Health Authorities (FDA and EMA) and SMA experts were had and the […]


  2. Novartis has acquired AveXis

    In April this year, we heard that talks were ongoing regarding a proposed acquisition of AveXis by Novartis. We have now been informed that this has happened so as that AveXis is now officially a Novartis company. AveXis has provided us with a community update, stating that the company remains steadfastly dedicated to the SMA community. […]


  3. Novartis expands Branaplam trial to new countries & other news

    Branaplam, formerly known as LMI070, an oral treatment under investigation for SMA, is currently in a Phase 1/2 clinical trial (safety and efficacy) in untreated SMA Type 1 patients. Novartis is pleased to announce a couple of developments: The expansion in the number of countries and sites for the second part of the trial. Specifically, in […]


  4. AveXis dosed first pre-symptomatic patient in SPR1NT trial

    AveXis, the gene therapy company developing a gene therapy to treat SMA, known as AVXS‐101, recently treated the first patient in a new study known as SPR1NT. SPR1NT is a multi-national study which will evaluate AVXS-101 in approximately 44 patients less than six weeks old with SMA Types 1, 2 or 3, who have 2, 3 […]


  5. Biogen releases statement on CHERISH data

    At our request, Biogen sent us a community update on the end of study results from CHERISH, the Phase 3 trial evaluating nusinersen for the treatment of individuals with later-onset SMA, which have now been published in the New England Journal of Medicine (NEJM). The full manuscript entitled, “Nusinersen Versus Sham Control in Later-Onset Spinal […]