An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
Titel:
An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study
Auteur:
Miller, Timothy M Pestronk, Alan David, William Rothstein, Jeffrey Simpson, Ericka Appel, Stanley H Andres, Patricia L Mahoney, Katy Allred, Peggy Alexander, Katie Ostrow, Lyle W Schoenfeld, David Macklin, Eric A Norris, Daniel A Manousakis, Georgios Crisp, Matthew Smith, Richard Bennett, C Frank Bishop, Kathie M Cudkowicz, Merit E