Thomas Sejersen, Sophie Graham, Anne-Berit Ekström, Anna-Karin Kroksmark, Marta Kwiatkowska, Michael L Ganz, Nahila Justo, Karl Gertow, Alex Simpson
BACKGROUND: Spinal muscular atrophy (SMA) is a rare, progressive, neuromuscular disorder. Recent advances in treatment require an updated assessment of burden to inform reimbursement decisions. OBJECTIVES: To quantify healthcare resource utilisation (HCRU) and cost of care for patients with SMA. METHODS: Cohort study of patients with SMA identified in the Swedish National Patient Registry (2007-2018), matched to a reference cohort grouped into four SMA types (1, 2, 3, unspecified adult onset [UAO])...
April 20, 2024: European Journal of Health Economics: HEPAC: Health Economics in Prevention and Care