Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

McDonald, CM; Campbell, C; Erazo Torricelli, R; Finkel, RS; Flanigan, KM; Goemans, N; Heydemann, P; ... Mercuri, E; + view all McDonald, CM; Campbell, C; Erazo Torricelli, R; Finkel, RS; Flanigan, KM; Goemans, N; Heydemann, P; Kaminska, A; Kirschner, J; Muntoni, F; Nascimento Osorio, A; Schara, U; Sejersen, T; Shieh, PB; Sweeney, HL; Topaloglu, H; Tulinius, M; Vilchez, JJ; Voit, T; Wong, B; Elfring, G; Kroger, H; Luo, X; McIntosh, J; Ong, T; Riebling, P; Souza, M; Spiegel, RJ; Peltz, SW; Mercuri, E; - view fewer (2017) Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet , 390 (10101) pp. 1489-1498. 10.1016/S0140-6736(17)31611-2 . Green open access

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