Article
Findings from the Longitudinal CINRG Becker Natural History Study.
Journal of neuromuscular diseases - 1 Jan 2024
Clemens Paula R, Gordish-Dressman Heather, Niizawa Gabriela, Gorni Ksenija, Guglieri Michela, Connolly Anne M, Wicklund Matthew, Bertorini Tulio, Mah Jean, Thangarajh Mathula, Smith Edward C, Kuntz Nancy L, McDonald Craig M, Henricson Erik, Upadhyayula S, Byrne Barry, Manousakis Georgios, Harper Amy, Iannaccone Susan, Dang Utkarsh J
Abstract excerpt
BACKGROUND: Becker muscular dystrophy is an X-linked, genetic disorder causing progressive degeneration of skeletal and cardiac muscle, with a widely variable phenotype. OBJECTIVE: A 3-year, longitudinal, prospective dataset contributed by patients with confirmed Becker muscular dystrophy was analyzed to characterize the natural history of this disorder. A better understanding of the natural history is crucial to...
Topics
- Adult
- Adolescent
- Humans
- Child
- Muscular Dystrophy, Duchenne
- Prospective Studies
- Cross-Sectional Studies
- Phenotype
- Myocardium
