Comparison of timed function test results in nmDMD patients receiving ataluren: STRIDE Registry vs phase 3 clinical trial

E. Mercuri, F. Muntoni, M. Tulinius, F. Buccella,I Desguerre,J. Kirschner,Osorio A. Nascimento,S. Johnson,C. Werner, J. Jiang,J. Li, J. Wing, P. Trifillis

ANNALS OF NEUROLOGY(2022)

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Neuromuscular Disorders, Rare Diseases
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