特集 神経疾患治療の進歩 2017
運動ニューロン疾患の治療の進歩
キーワード:
amyotrophic lateral sclerosis (ALS),
motor neuron disease (MND),
induced pluripotent stem cells (iPSCs),
novel therapy
2018 年 35 巻 5 号 p. 609-613
詳細
2018 年 35 巻 5 号 p. 609-613
Motor neuron diseases (MND) are neurodegenerative syndrome characterized by systemic and selective death of motor neurons. As a represetnative MND, amyotrophic lateral sclerosis (ALS) is the most rapidly progressive disease with adult–onset upper and lower motor neuron degeneration. This review provides an overview of preclinical and clinical advances in ALS research, and summarizes key literature on emerging therapeutic approaches in 2017. The topics include recently published data elucidating pathomechanisms by ALS–linked mutant SOD1, FUS, TARDBP, C9orf72, and recently reported genes. In addition, we describe novel therapeutics for MND, which refer to edaravone for ALS, leuprorelin for spinal bulbar muscular atrophy (SBMA), and nusinersen for spinal muscular atrophy (SMA).
