LAUSR

Amyotrophic lateral sclerosis (ALS) is a degenerative disease affecting the upper and lower motor neurons, leading to bulbar and spinal muscle weakness. Disease progression is more rapid than in most other neurodegenerative disorders, and patient prognosis is poor because most patients suffer from respiratory paresis in the advanced stages of the disease. Major genetic causes of ALS include mutations in SOD1 [1] and TARDBP [2], and an abnormal hexanucleotide repeat expansion in chromosome 9 open reading frame 72 (C9orf72) [3]. Despite extensive research into the molecular pathomechanism of ALS, clinical treatment options remain limited, with approved pharmacological treatment restricted to two drugs, riluzole and edaravone. Confirmative clinical trials of these drugs are shown in Table 1. Riluzole modulate glutamatergic neuronal transmission [4], and edaravone is a potent scavenger of reactive oxygen species [5].