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Journal article
Acsadi, G.; Shieh, P.; Crawford, T.; Richardson, R.; Natarajan, N.; Castro, D.; Gheuens, S.; Gambino, G.; Sun, P.; Reyna, S.; Farwell, W. und Müller-Felber, W.
(2017):
Safety and efficacy of nusinersen in infants/children with spinal muscular atrophy (SMA): part 1 of the phase 2 EMBRACE study.
In: Neuromuscular Disorders, Vol. 27: S210-S211