Abstract
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease primarily affecting motor neurons. Recently,1 Miller etal. presented phase 3 data for the RNase-H-recruiting antisense oligonucleotide (ASO) targeting superoxide dismutase 1 (SOD1) in ALS, which represents a step forward in the deployment of ASO therapeutics against CNS targets.
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