Economic evaluations of disease-modifying therapies for spinal muscular atrophy: a systematic literature review.

ABSTRACT:

Background

Spinal muscular atrophy (SMA) is a rare, life-limiting neuromuscular disorder characterised by progressive motor neuron degeneration. The recent emergence of disease-modifying therapies (DMTs), nusinersen, onasemnogene abeparvovec, and risdiplam, has revolutionised SMA care but presents economic challenges due to high treatment costs and limited long-term evidence.

Objective

To review and critically appraise economic evaluations that assessed the cost-effectiveness of DMTs in people living with SMA.

Methods

A systematic literature review was conducted following Cochrane and PRISMA guidelines. Initial searches were conducted in January 2024 and updated in February 2025. Searches were carried out in key biomedical and economic databases, as well as grey liter

SUBMITTER: Yousefi M 

PROVIDER: S-EPMC12797846 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature