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 provides update on splicing modifier RG7916

    RG7916Roche is planning to start clinical studies on RG7916 in people with SMA Type 1 and SMA Type 2/3 by the end of 2016 and are awaiting health authority feedback. This feedback will determine final countries, sites and timelines for the studies. An update will be provided in due course.

    Patients who participated in the Moonfish study will have the opportunity to enrol in an open label study with RG7916, if they fulfil study criteria and wish to participate. These patients will be contacted by their clinical trial site to update them on this development.

    RG7916 is a splicing modifier, a compound which works by increasing the amount of SMN protein made by the SMN2 gene.

    Please read the full community statement.