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.

Merck & Co. Inc. continued to broaden its reach in neurodegenerative diseases by paying, through a subsidiary, as much as $610 million to take over preclinical-stage Caraway Therapeutics Inc. The deal involves an undisclosed up-front payment along with contingent milestone rewards.

Amyotrophic lateral sclerosis (ALS) is a disorder that leads to progressive muscle weakness and loss of muscle control due to affectation of the motor neurons. Increasing evidence points to defects in the nuclear envelope, which leads to disease progression.

Fundamental Pharma GmbH has described TRPM4/NMDA interaction inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis (ALS).