Heart Transplantation Mortality in Recipients with Muscular Dystrophy
We want to compare mortality in heart transplant (HTx) recipients with and without Muscular Dystrophy.
UCLA researchers are part of an international team that reported the use of a stem cell gene therapy to treat nine people with the rare, inherited blood disease known as X-linked chronic granulomatous disease, or X-CGD. Six of those patients are now in remission and have stopped other treatments. Before now, people with X-CGD – which causes recurrent infections, prolonged hospitalizations for treatment, and a shortened lifespan – had to rely on bone marrow donations for a chance at remission.“With this gene therapy, you can use a patient’s own stem cells instead of donor cells for a transplant,&rdqu...
This study demonstrates for the first time that senescent cells secrete functional LTs, significantly contributing to the LTs pool known to cause or exacerbate idiopathic pulmonary fibrosis. Against Senolytics https://www.fightaging.org/archives/2019/11/against-senolytics/ There is no consensus in science that is so strong as to have no heretics. So here we have an interview with a naysayer on the matter of senolytic treatments, who argues that the loss of senescent cells in aged tissues will cause more harm to long-term health than the damage they will do by remaining. To be clear, I think this to be a ...
As survival and neuromuscular function in Duchenne Muscular Dystrophy (DMD) have improved with glucocorticoid therapy and advances in respiratory care, the proportion of cardiac deaths is increasing. Little is known about risk factors for cardiac causes of death in this population.
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.
There is variable practice and debate surrounding prophylactic heart failure (HF) therapy in Duchenne Muscular Dystrophy (DMD) patients with normal systolic function. We sought to determine the impact of HF medications including ACE inhibitors and ARB on the development of moderate systolic dysfunction or death in a cohort of DMD patients.
Current guidelines for the management of left ventricular systolic dysfunction (LVSD) in Duchenne Muscular Dystrophy (DMD) recommend angiotensin converting enzyme (ACE) inhibitors (class IIa). Little is known about the historic use of HF medications in Duchenne Muscular Dystrophy (DMD) and LVSD.
Little is known about sudden cardiac death risk and arrhythmias in boys with Duchenne muscular dystrophy(DMD). The goal of the study is to describe ECG findings and arrhythmia burden in a multi-center contemporary cohort of boys with DMD.
Duchenne muscular dystrophy (DMD) is characterized by myocardial fibrosis and left ventricular (LV) dysfunction. Implantable cardioverter defibrillator (ICD) use has not been characterized in this population but is generally considered for patients with severe LV dysfunction receiving goal directed medical therapy (GDMT), including a beta blocker plus an angiotensin converting enzyme inhibitor. We evaluated utilization and efficacy of ICD use in patients with DMD.
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Significant genetic heterogeneity exists in the dystrophin gene in males with Duchenne Muscular Dystrophy (DMD). The goal is to describe the impact of genetic variations on clinical outcomes.