Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy
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F. Metzger | S. Paushkin | L. Pellizzoni | S. Tisdale | F. Lotti | J. Narasimhan | Josephine Sheedy | A. Mollin | Shirley Yeh | M. Weetall | H. Ratni | G. Karp | N. Naryshkin | Karen S. Chen | A. Dakka | Xin Zhao | Zhihua Feng | C. Ko | J. Petruska | E. Welch | K. Ling