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Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany

Pechmann, Langer, Schorling, Stein, Vogt, Schara, Kölbel, Schwartz, Hahn, Giese, Johannsen, Denecke, Weiß, Theophil, Kirschner

J Neuromuscul Dis. 2018;5(2):135-143. doi: 10.3233/JND-180315.