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.

Alphamol Science Ltd. has divulged opioid receptor agonists reported to be useful for the treatment of pain.

Researchers from Neumirna Therapeutics ApS have presented an anti-miR-134 ASO approach named NMT.001 for the potential treatment of drug-resistant epilepsy.