News | SMA Europe - Part 34

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. ISIS launches open-label extension study SHINE

    Isis Pharmaceuticals announced the official launch of an open-label extension study, called SHINE, which will be continuing treatment of the same infants and children diagnosed with SMA who had previously participated in Phase III studies ENDEAR and CHERISH. The infants who participated in ENDEAR will continue to receive 12mg of ISIS-SMNRx every 4 months, while […]


  2. The role of the vascular system in SMA – Professor Simon Parson

    The University of Aberdeen has announced that researchers have made a significant breakthrough in understanding SMA. Funding for this research came in part from one of our member organisations, The SMA Trust. Vanessa Christie-Brown, Research Co-ordinator at The SMA Trust commented “We are delighted to have funded Professor Parson’s work on vascular involvement in SMA. […]


  3. Annual Cure SMA Conference – 16th to 19th June 2016 – Anaheim, California, USA

    The CURE-SMA annual conference is the largest SMA conference in the world. Through the conference, researchers, healthcare professionals and families are all brought together to network, learn and collaborate. The family conference includes a variety of workshops, keynote sessions with leading researchers, a family-friendly research poster session and more—plus fun events like the dance party, […]