Knopp is advancing novel agents to activate a key ion channel in nerve cells, targeting a devastating genetic mutation associated with neonatal encephalopathy, as well as epilepsy and neuropathic pain.
The mitochondrion is the powerhouse of the cell, providing energy to every cell type in the body. Brain cells rank among the most energy-demanding cells in the body. The disruption of cellular bioenergetic activity has emerged as a common factor in multiple pathways leading to nerve cell death in diseases such as ALS, Parkinson's and other neurodegenerative disorders.
Knopp's pipeline consists of investigational drug products that have not been approved by the U.S. Food and Drug Administration. These investigational drug products are still undergoing clinical study to verify their safety and effectiveness.