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. SMA Europe responds to European neuromuscular experts’ consensus statements on gene replacement therapy for SMA

    A group of European neuromuscular experts presented eleven consensus statements covering qualification, patient selection, safety considerations and long-term monitoring, in order to aid the rational use of Zolgensma™ for the treatment of SMA (1). In a letter published in the European Journal of Paediatric Neurology (2), SMA Europe addresses these consensus statements by highlights: The […]