Huntington’s disease is a multi-system disorder

Huntington’s disease (HD) is one ofthe most common non-curable rare diseases and is characterized by choreic movements, psychiatric symptoms, and slowly progressive dementia. HD is inherited as an autosomal dominant dis-order with complete penetrance. Although brain pathology has become a hallmark of HD, there is a critical mass of new studies suggesting peripheral tis-sue pathology as an important factor in disease progression. In particular, recently published studies about skeletal muscle malfunction and HD-related car-diomyopathy in HD mouse models strongly suggest their important roles, leading to upcoming preclinical and clin-ical trials. One might conclude that ther-apeutic approaches in HD should not be restricted only to the brain pathology but instead major efforts should also be made to understand the cross-talk between dis-eased tissues like the CNS-Heart or CNS-skeletal muscle axes