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Nature|April 24, 2007
PTC124 targets genetic disorders caused by nonsense mutationsEllen M Welch, Elisabeth R Barton, Jin Zhuo, et al.Science (New York, N.Y.)|August 9, 2014
Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophyNikolai A Naryshkin, Marla Weetall, Amal Dakka, et al.Pageof 5