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 community update on RG7916 for SMA

    Roche provides community updateRoche provides a community update on the investigational product RG7916, which they are developing together with PTC Therapeutics and the SMA Foundation in the US. This compound is an orally-administered SMN2 splicing modifier, for which there are 3 studies: Firefish, Sunfish and Jewelfish. All include different populations.

    They are pleased to report that Sunfish (Type 2 & 3) and Firefish (Type 1) are recruiting in Europe and will expand to include additional countries and sites in 2017. Jewelfish is expected to start recruiting in Europe and the US in early 2017.

    RG7916 works by making the “back-up” gene, SMN2, produce more functional SMN protein, which is essential for motor neuron survival.

    For more information on:

    Roche’s Community Update – December 2016

    The trials,  through Pioneering Healthcare website:

    Clinical trial sites and countries, through the Clinical Trials website: