A Decision for Life – Treatment decisions in newly diagnosed families with spinal muscular atrophy (SMA)


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 (Kirschner, et al., 2020). In response to this consensus statement, a group of patient representatives from SMA Europe highlight considerations that must be taken into account when considering treatment.

Authors: N. Gusset, Y. Erbas, O. Germanenko, K. Rucinski, E. Stumpe and M. de Lemus.

Journal: Neuromuscular Disorders

Link: 10.1016/j.ejpn.2020.11.003

New treatments for spinal muscular atrophy open-up new challenges for European & national institutions


SMA Europe discusses how new treatments for spinal muscular atrophy open up new challenges for European and national institutions

Authors: Marie-Christine Ouillade & Alexandre Méjat.


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Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients


Authors: F. Rouault, V. Christie-Brown, R. Broekgaarden, N. Gusset, D. Henderson, P. Marczuk, I. Schwersenz, G. Bellis, C. Cottet .

Journal: Neuromuscular Disorders
DOI: 10.1016/j.nmd.2017.01.018