2021 Volume 38 Issue 5 Pages 728-731
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 2020, positive results of several clinical trials including novel antisense oligonucleotide treatment on SOD1–ALS (tofersen) were published. As for SMA, novel gene–replacement treatment (zolgensma) was approved in Japan. These novel drugs will bring a promising future for MND patients.
This review provides an overview of clinical advances in MND research and summarizes selected key literature on therapeutic approaches in 2020.
