Location: US-New York
Drug Type: Protein Biologic
Mechanism Type: NMJ stabilization
Mechanism: This antibody-based approach aims to help muscle fibers and motor neurons stay connected in ALS by increasing the activity of the muscle-specific tyrosine kinase MuSK, a key enzyme that stabilizes neuromuscular junctions.
U.S. Status for ALS: Preclinical
 Preserving neuromuscular synapses in ALS by stimulating MuSK with a therapeutic agonist antibody. Cantor S. et al. Elife. 2018 Feb 20;7
 Increasing MuSK activity delays denervation and improves motor function in ALS mice. Pérez-García MJ, Burden SJ. Cell Rep. 2012 Sep 27;2(3):497-502.