Disease modifying agents for Huntington's disease - What is on the horizon?

Huntington's disease is an autosomal dominant neurodegenerative disorder ch aracterised by abnormalities of motor function, cognition and behaviour. Th e striatum is particularly vulnerable to the disease process with selective loss of medium-sized spiny projection neurons and relative sparing of inte rneurons. Although the underlying mechanism initiating and propagating neuronal destr uction is currently unknown, experimental evidence over the past decade has implicated mitochondrial dysfunction and excitotoxicity in the pathogenesi s of Huntington's disease. The current treatment of Huntington's disease is limited to palliative care with secondary symptomatic management. Recent a dvances in the understanding of the pathophysiology of this disease may lea d to the development of pharmacotherapeutic and neurosurgical interventions to effectively treat this devastating disease.