DOI:
10.1055/s-00000140
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References
McDonald CM, Campbell C, Torricelli RE. et al.
Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.
Lancet 2017;
390: 1489-1498
doi:10.1016/S0140-6736(17)31611-2
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