The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review.

ABSTRACT:

Background

Duchenne muscular dystrophy (DMD) is a severe rare progressive inherited neuromuscular disorder, leading to loss of ambulation (LOA) and premature mortality. The standard of care for patients with DMD has been treatment with corticosteroids for the past decade; however a synthesis of contemporary data describing the clinical course of DMD is lacking. The objective was to summarize age at key clinical milestones (loss of ambulation, scoliosis, ventilation, cardiomyopathy, and mortality) in the corticosteroid-treatment-era.

Methods

A systematic review was conducted using MEDLINE and EMBASE. The percentage experiencing key clinical milestones, and the mean or median age at those milestones, was synthesized from studies from North American populations, published betwe

SUBMITTER: Szabo SM 

PROVIDER: S-EPMC8141220 | biostudies-literature | 2021 May

REPOSITORIES: biostudies-literature