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ABSTRACT: 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]). HCRU included inpatient admissions, outpatient visits, procedures, and dispensed medications. Direct medical costs were estimated by multiplying HCRU by respective unit costs. Average annual HCRU and medical costs were modelled for SMA versus reference cohorts to estimate dif
SUBMITTER: Sejersen T
PROVIDER: S-EPMC11743398 | biostudies-literature | 2025 Feb
REPOSITORIES: biostudies-literature