Treatment of dystrophin cardiomyopathies

J Finsterer, L Cripe - Nature Reviews Cardiology, 2014 - nature.com
Abstract Treatment of cardiac disease in patients with dystrophinopathies substantially
improves outcomes. In this Review, we summarize and discuss findings from the past 20 …

Dystrophin-deficient cardiomyopathy

F Kamdar, DJ Garry - Journal of the American College of Cardiology, 2016 - jacc.org
Dystrophinopathies are a group of distinct neuromuscular diseases that result from
mutations in the structural cytoskeletal Dystrophin gene. Dystrophinopathies include …

Evolving therapeutic strategies for dystrophinopathies: potential for conflict between cardiac and skeletal needs

SD Colan - Circulation, 2005 - Am Heart Assoc
The importance of cardiomyopathy to the clinical course of the dystrophinopathies varies
according to the nature of the dystrophin defect. In Becker muscular dystrophy (BMD) …

The heart in human dystrophinopathies

J Finsterer, C Stöllberger - Cardiology, 2003 - karger.com
Dystrophinopathies are due to mutations in the dystrophin gene on chromosome Xp21. 1
and comprise the allelic entities Duchenne muscular dystrophy (DMD), Becker muscular …

[HTML][HTML] Cardiac involvement in dystrophin-deficient females: current understanding and implications for the treatment of dystrophinopathies

KRQ Lim, N Sheri, Q Nguyen, T Yokota - Genes, 2020 - mdpi.com
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive condition caused
primarily by out-of-frame mutations in the dystrophin gene. In males, DMD presents with …

[HTML][HTML] Dystrophin cardiomyopathies: clinical management, molecular pathogenesis and evolution towards precision medicine

D D'Amario, A Gowran, F Canonico… - Journal of clinical …, 2018 - mdpi.com
Duchenne's muscular dystrophy is an X-linked neuromuscular disease that manifests as
muscle atrophy and cardiomyopathy in young boys. However, a considerable percentage of …

New approaches in the therapy of cardiomyopathy in muscular dystrophy

EM McNally - Annu. Rev. Med., 2007 - annualreviews.org
Cardiomyopathy is a frequent occurrence in muscular dystrophy, and heart disease in
muscular dystrophy can contribute to both morbidity and mortality. A number of novel …

Prevalence and clinical outcomes of dystrophin‐associated dilated cardiomyopathy without severe skeletal myopathy

MA Restrepo‐Cordoba, K Wahbi… - European journal of …, 2021 - Wiley Online Library
Aims Dilated cardiomyopathy (DCM) associated with dystrophin gene (DMD) mutations in
individuals with mild or absent skeletal myopathy is often indistinguishable from other DCM …

[HTML][HTML] Dystrophic cardiomyopathy: complex pathobiological processes to generate clinical phenotype

T Tsuda, KK Fitzgerald - Journal of cardiovascular development and …, 2017 - mdpi.com
Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and X-linked
dilated cardiomyopathy (XL-DCM) consist of a unique clinical entity, the dystrophinopathies …

Pathophysiology and therapy of cardiac dysfunction in Duchenne muscular dystrophy

DP Judge, DA Kass, WR Thompson… - American Journal of …, 2011 - Springer
Cardiac dysfunction is a frequent manifestation of Duchenne muscular dystrophy (DMD) and
a common cause of death for individuals with this condition. Early diastolic dysfunction and …