Pharmacokinetics, pharmacodynamics, and efficacy of a small-molecule SMN2 splicing modifier in mouse models of spinal muscular atrophy.

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作者： Xin,Zhao ^[1] ; Zhihua,Feng ^[2] ; Karen K Y,Ling ^[2] ; Anna,Mollin ^[1] ; Josephine,Sheedy ^[1] ; Shirley,Yeh ^[1] ; Janet,Petruska ^[1] ; Jana,Narasimhan ^[1] ; Amal,Dakka ^[1] ; Ellen M,Welch ^[1] ; Gary,Karp ^[1] ; Karen S,Chen ^[3] ; Friedrich,Metzger ^[4] ; Hasane,Ratni ^[4] ; Francesco,Lotti ^[5] ; Sarah,Tisdale ^[5] ; Nikolai A,Naryshkin ^[1] ; Livio,Pellizzoni ^[5] ; Sergey,Paushkin ^[3] ; Chien-Ping,Ko ^[6] ; Marla,Weetall ^[7]

作者单位： PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA. ^[1] Department of Biological Sciences, Section of Neurobiology, University of Southern California, Los Angeles, CA 90089, USA. ^[2] SMA Foundation, 888 Seventh Avenue, Suite 400, New York, NY 10019, USA. ^[3] F. Hoffmann-La Roche, Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Grenzacherstrasse 124, Basel 4070, Switzerland. ^[4] Department of Pathology and Cell Biology, Center for Motor Neuron Biology and Disease, Columbia University, New York, NY 10032, USA and. ^[5] Department of Biological Sciences, Section of Neurobiology, University of Southern California, Los Angeles, CA 90089, USA, mweetall@ptcbio.com cko@usc.edu. ^[6] PTC Therapeutics, Inc., South Plainfield, NJ 07080, USA, mweetall@ptcbio.com cko@usc.edu. ^[7]