CO177 The Burden of Spinal Muscular Atrophy Type 1 on Caregivers in Argentina: First Results of a Global Survey Launched in Latin America

Abstract

Spinal muscular atrophy type 1 (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. Data on caregiver burden associated with SMA1 are sparse. We sought to describe sociodemographic and time costs for families and caregivers of patients with SMA1. Here, we present initial data reflecting caregiver burden in Argentina.