An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: A phase 1, randomised, first-in-man study

Timothy M. Miller, Alan Pestronk, William David, Jeffrey Rothstein, Ericka Simpson, Stanley H. Appel, Patricia L. Andres, Katy Mahoney, Peggy Allred, Katie Alexander, Lyle W. Ostrow, David Schoenfeld, Eric A. Macklin, Daniel A. Norris, Georgios Manousakis, Matthew Crisp, Richard Smith, C. Frank Bennett, Kathie M. Bishop, Merit E. Cudkowicz

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