Burden and Treatment of Achondroplasia: A Systematic Literature Review.

ABSTRACT:

Background

Achondroplasia is the most common form of skeletal dysplasia. Recent advances in therapeutic options have highlighted the need for understanding the burden and treatment landscape of the condition. This systematic literature review (SLR) aimed to identify health-related quality of life (HRQoL)/utilities, healthcare resource use (HCRU), costs, efficacy, safety and economic evaluation data in achondroplasia and to identify gaps in the research.

Methods

Searches of MEDLINE, Embase, the University of York Centre for Reviews and Dissemination (CRD), the Cochrane Library and grey literature were performed. Articles were screened against pre-specified eligibility criteria by two individuals and study quality was assessed using published checklists. Additional targeted se

SUBMITTER: Murton MC 

PROVIDER: S-EPMC10427595 | biostudies-literature | 2023 Sep

REPOSITORIES: biostudies-literature