PCR61 The Burden of Spinal Muscular Atrophy Type 2 (SMA2) on Caregivers in Japan, United Kingdom, and United States: Results of a Global Survey

Abstract

SMA2, a rare, genetic neuromuscular disease that causes loss of both voluntary motor and bulbar functions, accounts for 40–50% of all SMA cases worldwide. Because data on caregiver burden associated with SMA2 are sparse, we sought to describe out-of-pocket expenditures and time costs for the families/caregivers of these patients, as well as health care resource utilization (HCRU). Here, we present initial data reflecting caregiver burden in Japan, UK, and US.