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- [41] An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study LANCET NEUROLOGY, 2013, 12 (05): : 435 - 442
- [48] Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS NEW ENGLAND JOURNAL OF MEDICINE, 2020, 383 (02): : 109 - 119