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. RG7916 community update from Roche

    Roche has shared with us an update of the RG7916, now called risdiplam, programme, which has reached a number of milestones. Risdiplam is an SMN2 splicing modifier which is given daily by mouth or by g-tube as a liquid and distributes widely throughout the body. Risdiplam has been designed to help the SMN2 gene produce more […]


  2. 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 […]


  3. 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. […]


  4. 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 […]