sma carni-val trial part i double-blind, randomized, placebo-controlled trial of l-carnitine and valproic acid in spinal muscular atrophysma carni-val试验部分我双盲,随机,安慰剂对照试验左卡尼汀和丙戊酸的脊髓性肌萎缩.pdf

SMA CARNI-VAL Trial Part I: Double-Blind, Randomized, Placebo-Controlled Trial of L-Carnitine and Valproic Acid in Spinal Muscular Atrophy 1 2 4 3 1 Kathryn J. Swoboda *, Charles B. Scott , Thomas O. Crawford , Louise R. Simard , Sandra P. Reyna , 10 9 14 12 11 Kristin J. Krosschell , Gyula Acsadi , Bakri Elsheik , Mary K. Schroth , Guy D’Anjou , Bernard 5 6 7 8 1 LaSalle , Thomas W. Prior , Susan L. Sorenson , Jo Anne Maczulski , Mark B. Bromberg , Gary M. Chan13, John T. Kissel14 for the Project Cure Spinal Muscular Atrophy Investigators Network 1 Department of Neurology, University of Utah School of Medicine, Salt Lake City, Utah, United States of America, 2 CBS Squared, Inc, Fort Washington, Pennsylvania, United States of America, 3 Department of Biochemistry and Medical Genetics, University of Manitoba, Winnipeg, Manitoba, Canada, 4 Departments of Neurology and Pediatrics, Johns Hopkins University School of Medicine, Baltimore, Maryland, United States of America, 5 General Clinical Research Center, University of Utah School of Medicine, Salt Lake City, Utah, United States of America, 6 Department of Molecular Pathology, Ohio State University, Columbus, Ohio, United States of America, 7 Primary Children’s Medical Center, Salt Lake City, Utah, United States of America, 8 Pediatric Occupational Therapy Services, Chicago, Illinois, United States of America, 9 Departments of Neurology and Pediatrics, Wayne State University School of Medicine, Detroit, Michigan, United States of