Long-term tofersen in SOD1 amyotrophic lateral sclerosis
T.M. Miller, M.E. Cudkowicz, P.J. Shaw, A. Genge et autres
Importance Approximately 2% of amyotrophic lateral sclerosis (ALS) cases are attributable to a pathogenic variant in the superoxide dismutase 1 (SOD1) gene. Tofersen, an intrathecal antisense oligonucleotide designed to reduce SOD1 protein synthesis, is the first and only approved therapy for the …
us, gb, jp (code pays fourni par la source)
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White Rose Research Online (University of Leeds, The University of Sheffield, University of York)