Characterizing the Occurrence of Key Clinical Milestones in Duchenne Muscular Dystrophy in the United States Using Real-World Data.

ABSTRACT:

Background

Data on the clinical course of Duchenne muscular dystrophy (DMD) exist from well-characterized clinical cohorts but estimates from real-world populations are fewer.

Objective

The objective was to estimate the prevalence of key clinical milestones by age, among real-world commercially-insured DMD patients in the United States.

Methods

MarketScan claims (2013-2018) were used to identify males with DMD. The percentages with wheelchair use or experiencing scoliosis, neurologic/neuropsychiatric involvement, cardiomyopathy, and respiratory involvement were tabulated; as were the median (interquartile range [IQR]) ages at first observed occurrence within the claims data.

Results

Among DMD patients (n = 1,964), the median (IQR) baseline age was 15 (9-21) yea

SUBMITTER: Szabo SM 

PROVIDER: S-EPMC9697036 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature