Sources of variation in estimates of Duchenne and Becker muscular dystrophy prevalence in the United States.

ABSTRACT:

Background

Direct estimates of rare disease prevalence from public health surveillance may only be available in a few catchment areas. Understanding variation among observed prevalence can inform estimates of prevalence in other locations. The Muscular Dystrophy Surveillance, Tracking, and Research Network (MD STARnet) conducts population-based surveillance of major muscular dystrophies in selected areas of the United States. We identified sources of variation in prevalence estimates of Duchenne and Becker muscular dystrophy (DBMD) within MD STARnet from published literature and a survey of MD STARnet investigators, then developed a logic model of the relationships between the sources of variation and estimated prevalence.

Results

The 17 identified sources of variability fel

SUBMITTER: Whitehead N 

PROVIDER: S-EPMC10031951 | biostudies-literature | 2023 Mar

REPOSITORIES: biostudies-literature