Key result
This comprehensive overview discusses the pathophysiology and management of Becker muscular dystrophy, highlighting that cardiomyopathy is the leading cause of death and exploring new gene therapies.
This review provides a comprehensive overview of the pathophysiology and management of cardiomyopathy in Becker muscular dystrophy, including established treatments and emerging gene therapies.
Answers are grounded in this paper and cite their sources.
Cardiomyopathy surveillance remains essential in Becker muscular dystrophy; leaves open the clinical translation of emerging gene therapies.
Core tip: Becker muscular dystrophy (BMD) is an X-linked recessive disorder involving mutations of the dystrophin gene. This condition is rare but not uncommon. However, there are limited articles on this topic. Patients with BMD can present with mental retardation and diffuse muscular dystrophy. Cardiomyopathy is the number one cause of death in BMD. This paper aims to provide a comprehensive overview of BMD pathophysiology and management. The paper will discuss both the established treatments as well as exciting new research on gene therapy.
No takes yet. Share an insight, caveat, or question.
Ho et al. (2016) conducted a review in Becker muscular dystrophy and cardiomyopathy. Established treatments and gene therapy was evaluated. This comprehensive overview discusses the pathophysiology and management of Becker muscular dystrophy, highlighting that cardiomyopathy is the leading cause of death and exploring new gene therapies.
Synapse has enriched 10 related papers. Consider them for comparative context: