Direct Medical Costs of Spinal Muscular Atrophy in the Catalonia Region: A Population-Based Analysis

Abstract

Background : Spinal muscular atrophy (SMA) is a rare disorder, estimated to affect 1 per 10,000 live births. Patients affected with SMA often require intensive, chronic healthcare, which represents great social and economic costs. / Objective : This study aimed to evaluate the direct medical costs of SMA, from the National Health System perspective in Catalonia, and provide regional data for the development of optimal disease management protocols and resource allocation decisions at the regional level (...)