Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level.

Leal Therapeutics Inc. has divulged mitochondrial glutaminase kidney isoform (GLS, GLS1) inhibitors reported to be useful for the treatment of neurological and psychiatric disorders.