Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy.

ABSTRACT:

Introduction

The recent advent of disease-modifying therapies (DMTs) has dramatically changed the treatment landscape of spinal muscular atrophy (SMA), and the multifaceted impact of this advancement has not been assessed thoroughly in the growing body of literature. We sought to summarize the literature on the natural history of SMA and the impact of SMA DMTs, including health-related quality of life (HRQOL) and utilities, clinical efficacy and safety, and economic impact.

Methods

Systematic literature reviews were conducted following PRISMA guidelines with no inclusive dates. Relevant studies were identified by searching full-text databases on November 12-13, 2020, including MEDLINE, EMBASE, the Cochrane Central Register of Controlled Trials, and EconLit, conference procee

SUBMITTER: Yang M 

PROVIDER: S-EPMC9056474 | biostudies-literature | 2022 May

REPOSITORIES: biostudies-literature