Targeting Cellular Pathways in Severe Neurological Disorders: Advanced Chemical Biology Strategies for Alternating Hemiplegia of Childhood (AHC) and Cerebro-Oculo-Fascio-Skeletal (COFS) Syndrome Potential Effective Treatment
The main aim of this paper is to deeply investigate and highlight novel and innovative combined therapeutic strategies—such as potent anti-inflammatory and antioxidant molecules, small-molecule stabilizers, gene therapy, and pathway-specific inhibitors - targeting and restoring cellular homeostasis.