AVXS-101 phase I gene therapy clinical trial in SMA Type 1
J. R. Mendell,S. Al-Zaidy,R. Shell,W. D. Arnold,L. Rodino-Klapac,T. W. Prior,C. Miranda,L. Lowes,L. Alfano,K. Berry,K. Church,J. T. Kissel,S. Nagendran,D. M. Sproule, J. A. Cardenas,A. H. Burghes,K. D. Foust,K. Meyer, S. Likhite,B. K. Kaspar HUMAN GENE THERAPY(2016)
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