POSC158 The Burden of Spinal Muscular Atrophy Type 1 (SMA1) on Caregivers in Japan: First Results of a Global Survey

Abstract

SMA1, a rare, genetic neuromuscular disease that causes loss of both voluntary motor and bulbar functions, is usually fatal by 2 years of age if untreated. As data on caregiver burden associated with SMA1 are sparse, we sought to describe out-of-pocket expenditures and time costs for the families/caregivers of these patients, as well as healthcare resource use (HCRU). Here, we present initial data reflecting caregiver burden in Japan.