Challenges and Opportunities in Atypical Parkinsonian Syndromes: Call to Action

摘要

Neurodegenerative diseases are a leading cause of disability and death in the United States, and the burden of rare neurodegenerative conditions such as atypical Parkinsonian syndromes is expected to grow in the coming years as the size of the older population increases at an unprecedented rate. Atypical Parkinsonian syndromes encompass a collective of rare neurodegenerative diseases that are often misdiagnosed as Parkinson’s disease due to similar signs and symptoms. These syndromes include progressive supranuclear palsy (PSP), multiple system atrophy (MSA), and corticobasal degeneration (CBD), and are characterized by rapid disease progression and decreased life expectancy. In this paper, we describe the substantial burden atypical Parkinsonian syndromes pose to the healthcare system and patients attributable to challenges in diagnosis, ineffective treatment options, and rapid functional decline. Despite increased understanding and recognition of these disorders, there remains significant unmet need for patients with atypical Parkinsonian syndromes. We provide recommendations to policymakers to support access to effective disease management of atypical Parkinsonian syndromes through legislative efforts that i) prioritize development of disease-modifying treatments, ii) focus on objectively assessing disease progression in addition to symptom management, and iii) bring forth economic frameworks that capture the full value of treatments.