2022 年 39 巻 5 号 p. 778-781
Motor neuron diseases (MND) are devastating neurodegenerative disorder which primary affects motor neurons : amyotrophic lateral sclerosis (ALS), spinal bulbar muscular atrophy (SBMA), and spinal muscular atrophy (SMA). In 2021, results of open–label extension study of sodium phenylbutyrate–taurursodiol and long–term observational study of masitinib were published, which both studies proved the positive effects for survival of ALS. As for SMA, risdiplam was approved in Japan. And 5 years observational study of onasemnogene abeparvovec showed lasting effect.
This review provides an overview of clinical advances in MND research and summarizes selected key literature on therapeutic approaches in 2021.
