Previous research has demonstrated that systemic administration of a P2X7 agonist improved motor performance in a mouse model of amyotrophic lateral sclerosis (ALS) through enhancing satellite cells and the muscle pro-regenerative activity of infiltrating macrophages.

Axonal degeneration is a key early pathogenic driver of amyotrophic lateral sclerosis and other neurodegenerative diseases. Activation of calpain-2 (CAPN2) is thought to be the critical effector behind axonal degeneration. Amylyx Pharmaceuticals Inc. has presented data on their CAPN2 inhibitor antisense oligonucleotide (ASO) AMX-0114 as a potential therapeutic for axonal degeneration.

Researchers have defined an amyotrophic lateral sclerosis (ALS) reversal phenotype as having an initial diagnosis of ALS but subsequently showing a progressive and sustained clinical improvement, based on an unusual case they found.