A protein could be key to preserving heart function in Duchenne muscular dystrophy
(American Heart Association) The brain-derived neurotrophic factor protein, known to be vital for brain function, might hold the key for preserving heart function in children and young adults with Duchenne muscular dystrophy.Heart failure often kills Duchenne patients early in life and there is no effective treatment.Developing therapies designed to activate or supplement brain-derived neurotrophic factor could give new hope to patients at risk for Duchenne cardiomyopathy.
Patients with muscular dystrophy may have an associated cardiomyopathy, resulting in advanced heart failure requiring transplantation. However, the skeletal muscle dysfunction can result in respiratory impairment, dysphagia, and inability to participate in rehabilitation after heart transplantation. The purpose of this study was to describe the characteristics and outcomes of muscular dystrophy patients undergoing heart transplantation at our center.
AbstractThe longer survival of patients with Duchenne muscular dystrophy due to advances in clinical care has increased the incidence of Duchenne muscular dystrophy-associated cardiomyopathy, a nearly consistent feature in the third decade of life. A 26-year-old patient with Duchenne muscular dystrophy experienced severe acute heart failure triggered by pneumonia. Levosimendan was effective in improving heart function.
(Children's National Health System) Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects. Eplerenone, a heart failure drug, is used in older patients to treat cardiomyopathy, a leading cause of mortality for people with DMD. A new medicine under development appears to combine the beneficial effects of these drugs for the heart and muscle while also showing improved safety in experimental models.
Duchenne muscular dystrophy (DMD) is an X-linked disorder affecting 1 in 4700 male births.1 Although perceived primarily as a skeletal myopathy, boys also develop insidious and progressive cardiomyopathy. In the current era, cardiomyopathy is the leading cause of mortality.2 Because of skeletal muscle weakness, boys with cardiomyopathy are usually asymptomatic until they develop severe left ventricular (LV) dysfunction. Cardiac imaging is the primary modality for diagnosis of dysfunction. Unfortunately, standard heart failure biomarkers, such as brain natriuretic peptide (BNP), are only increased at end stage.
Conclusions: Even though the BGP-15-treatment did not interfere significantly with glucose homeostasis and vascular status, it considerably enhanced diastolic function, by affecting the SERCA/phospholamban pathway in GotoK rats. Although it requires further investigation, BGP-15 may offer a new therapeutic approach in DCM.
This article is protected by copyright. All rights reserved. PMID: 30674086 [PubMed - as supplied by publisher]
Duchenne muscular dystrophy (DMD) is an X-linked neuromuscular condition caused by mutations in the dystrophin gene leading to skeletal muscle weakness and dilated cardiomyopathy. The prevalence of DMD-related cardiomyopathy increases with age and is almost universal by the third decade of life. Myocardial fibrosis and progressive left ventricular dysfunction lead to the development of heart failure and premature death. With modern advances in medical and surgical management for patients with DMD increasing their life expectancy, cardiac dysfunction represents an increasing cause of morbidity and mortality in these patient...
In conclusion, this study demonstrated that allogeneic WT-MPC-Exo transplantation transiently restored dystrophin gene expression and improved cardiac function in MDX mice, suggesting that allogenic exosomal delivery may serve as an alternative treatment for cardiomyopathy of DMD.
Duchenne muscular dystrophy (DMD) is a systemic X-linked disorder with an incidence of 10.7 to 27.8 per 100,000 . The disease results from a mutation in the gene for the dystrophin protein resulting in progressive muscle necrosis, apoptosis and fibrosis. Muscle cell destruction directly affects many organ systems resulting in loss of ambulation, respiratory failure, heart failure and premature death [2,3]. With improvements in respiratory management, cardiac disease has emerged as a major cause of morbidity and mortality rendering the cardiologist an essential member of the multidisciplinary DMD care team .