An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
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A. Pestronk | D. Schoenfeld | P. Andres | J. Rothstein | M. Cudkowicz | C. Bennett | S. Appel | D. Norris | K. Bishop | T. Miller | E. Simpson | L. Ostrow | G. Manousakis | E. Macklin | Richard Smith | W. David | K. Mahoney | P. Allred | K. Alexander | Matthew J. Crisp | Daniel A. Norris | Stanley H. Appel | Timothy M Miller | Patricia L Andres | Eric A Macklin | C Frank Bennett | Timothy M. Miller