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Pharmacokinetics, pharmacodynamics, and efficacy of a small-moleculeSMN2splicing modifier in mouse models of spinal muscular atrophy

Journal Article published 29 Feb 2016 in Human Molecular Genetics volume 25 issue 10 on pages 1885 to 1899

Research funded by Spinal Muscular Atrophy Foundation | National Institute of Neurological Disorders and Stroke (R01NS069601)

Authors: Xin Zhao, Zhihua Feng, Karen K. Y. Ling, Anna Mollin, Josephine Sheedy, Shirley Yeh, Janet Petruska, Jana Narasimhan, Amal Dakka, Ellen M. Welch, Gary Karp, Karen S. Chen, Friedrich Metzger, Hasane Ratni, Francesco Lotti, Sarah Tisdale, Nikolai A. Naryshkin, Livio Pellizzoni, Sergey Paushkin, Chien-Ping Ko, Marla Weetall