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.
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