運動ニューロン疾患の治療の進歩

抄録

Motor neuron diseases (MND) are devastating neurodegenerative disorders that primarily affect motor neurons, including amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and spinal bulbar muscular atrophy (SBMA). In 2023, the FDA approved tofersen, an antisense nucleotide targeting the SOD1 mutation, based on results from an open–label extension of VALOR trial and the observed reduction in neurofilament light chain levels in plasma, despite the VALOR trial not reaching its primary endpoint. This review provides a comprehensive overview of the clinical advancements in MND research and summarizes key literature on therapeutic approaches from 2023.