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. AveXis completes phase 1 clinical trial enrollment

    AveXis has announced that they have completed enrollment in a Phase 1 study testing systemic delivery of gene therapy product AVXS-101, for spinal muscular atrophy. The open-label, dose-escalation study is designed to evaluate safety and preliminary indications of efficacy of AVXS-101 in patients with SMA Type 1. The primary outcome in the study is safety and […]


  2. Cytokinetics initiates a phase 2 clinical trial of CK-2127107

    Cytokinetics has initiated enrollment for a Phase 2 study testing CK-2127107 in teens and adults with SMA type II, III or IV, in California, USA. The clinical trial is designed to assess the effect of CK-2127107 on multiple measures of muscle function in both ambulatory and non-ambulatory patients with SMA. In collaboration with Astellas, Cytokinetics is […]


  3. Isis Pharmaceuticals changes its name to Ionis Pharmaceuticals

    Isis Pharmaceuticals, Inc. today announced that the company has changed its name to Ionis Pharmaceuticals, Inc. Ionis Pharmaceuticals is an original name that the Company has chosen to represent its innovative culture and heritage as both the pioneer and leader in the RNA-targeted therapeutic space for the past 26 years. Ionis is focused on bringing […]


  4. 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 […]