Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy by Finkel, RS, Mercuri, E, Darras, BT, Connolly, AM, Kuntz, NL, Kirschner, J, Chiriboga, CA, Saito, K, Laurent Servais, Tizzano, E, Topaloglu, H, Tulinius, M, Montes, J, Glanzman, AG, Bishop, K, Zhong, ZJ, Gheuens, S, Bennett, CF, Schneider, E, Farwell, W, De Vivo, DC, ENDEAR Study Group

Nusinersen versus sham control in later-onset spinal muscular atrophy by Mercuri, E, Darras, BT, Chiriboga, CA, Day, JW, Campbell, C, Connolly, AM, Iannaccone, ST, Kirschner, J, Kuntz, NL, Saito, K, Shieh, PB, Tulinius, M, Mazzone, ES, Montes, J, Bishop, KM, Yang, Q, Foster, R, Gheuens, S, Bennett, CF, Farwell, W, Schneider, E, De Vivo, DC, Finkel, RS

X-linked myotubular myopathy: A prospective international natural history study by Servais, L, Annoussamy, M, Lilien, C, Gidaro, T, Gargaun, E, Chê, V, Schara, U, Gangfuß, A, D'Amico, A, Dowling, JJ, Darras, BT, Daron, A, Hernandez, A, de Lattre, C, Arnal, J-M, Mayer, M, Cuisset, J-M, Vuillerot, C, Fontaine, S, Bellance, R, Biancalana, V, Buj-Bello, A, Hogrel, J-Y, Landy, H