Targeting Ferroptosis in Rare Neurological Disorders Including Pediatric Conditions: Innovations and Therapeutic Challenges

Ferroptosis, characterized by iron dependency and lipid peroxidation, has emerged as a key mechanism underlying neurodegeneration in rare neurological disorders. These conditions, often marked by significant therapeutic gaps and high unmet medical needs, present unique challenges for intervention development. This review examines the involvement of ferroptosis in rare neurological disease pathogenesis, focusing on its role in oxidative damage and neuronal dysfunction. We explore recent pharmacological advancements, including iron chelators, lipid peroxidation blockers, and antioxidant-based strategies, designed to target ferroptosis. While these approaches show promise, challenges such as disease heterogeneity, limited diagnostic tools, and small patient cohorts hinder progress. Furthermore, we discuss the translational and regulatory barriers to implementing ferroptosis-based therapies in clinical practice. By addressing these obstacles and fostering innovative solutions, this review underscores the potential of ferroptosis-targeting strategies to revolutionize treatment paradigms for rare neurological disorders.