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SMN2 splice modulators enhance U1-pre-mRNA association and rescue SMA mice.
Nature Chemical Biology, 2015 J. Palacino, Susanne E. Swalley, Cheng Song, Atwood K Cheung, Lei Shu, Xiaolu Zhang, Mailin Van Hoosear, Y. Shin, D. Chin, C. Keller, M. Beibel, N. Renaud, Thomas M. Smith, M. Salcius, Xiaoying Shi, M. Hild, Rebecca Servais, Monish Jain, Lin Deng, Caroline Bullock, Michael McLellan, S. Schuierer, Leo Murphy, M. Blommers, Cecile Blaustein, Frada Berenshteyn, Arnaud Lacoste, J. Thomas, G. Roma, G. Michaud, B. Tseng, J. A. Porter, V. Myer, John A. Tallarico, L. Hamann, D. Curtis, M. Fishman, W. Dietrich, N. Dales, Rajeev Sivasankaran +39 moresemanticscholar +1 more sourceNeurofilament light chain in serum of adolescent and adult SMA patients under treatment with nusinersen
Journal of Neurology, 2019 C. Wurster, P. Steinacker, R. Günther, J. Koch, P. Lingor, Z. Uzelac, S. Witzel, K. Wollinsky, B. Winter, Alma Osmanovic, O. Schreiber-Katz, Rami Al Shweiki, A. Ludolph, S. Petri, A. Hermann, M. Otto +15 moresemanticscholar +1 more source