The area where the motor neurons and muscles "connect" is called the neuromuscular junction (NMJ). NMJ health and function is a crucial part of the nervous system and highly scrutinized in ALS research. This area has proven itself promising with respect to targeting treatments aimed at stabilizing the connection or even re-establishing lost connections between existing motor neuron and muscle tissue in ALS patients.
We are currently conducting pharmacokinetic and pharmacodynamic experiments to determine an appropriate dosing stratagem to move this project into later-stage preclinical testing, such as survival efficacy screening.
Topics in the Pipeline