Duchenne Muscular Dystrophy (DMD): overview and pathomechanismFerlini, AlessandraEuropean Journal of Translational & Clinical Medicine
Duchenne muscular dystrophy: an overview to the cardiologistDuchenne muscular dystrophycardiac evaluationdilated cardiomyopathymuscular dystrophyIntroduction: Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in children, affecting approximately one in 3,500-5,000 liveborn boys....
Duan, D. Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy. Mol. Ther. 26, 2337–2356 (2018). Comprehensive overview of microdystrophin gene therapy development, its opportunities and challenges. Article CAS PubMed PubMed Central Google Scholar Bradley, W. G., Hudgson,...
Duchenne muscular dystrophy is the most common form of dystrophies. It is also the first genetic disease for which the responsible gene was identified through positional cloning. However, there still is no cure for this devastating disease. By inhibiting the elevation in calcium concentrations...
ACE = angiotensin-converting enzyme; DMD = Duchenne muscular dystrophy; EU = European Union; FDA = Food and Drug Administration; HF = heart failure; LVEF = left ventricular ejection fraction. Noncardiac Therapeutic Strategies Over the past several decades, several therapeutic advances have led to ...
Duchenne Muscular Dystrophy: Mechanism of Disease This brief video provides an overview of Duchenne muscular dystrophy. Learn about the signs and symptoms of Duchenne, how it is inherited, the functional role of dystrophin, and the biomolecular basis for the disease. ...
Duchenne Muscular Dystrophy Overview Duchenne muscular dystrophy (DMD) is referred to as a genetic disorder that is characterized by progressive muscle degeneration and weakness owing to alterations of protein called dystrophin that aids in keeping muscles intact. DMD is one amongst four conditions called...
Duchenne muscular dystrophy (DMD) is a severe, progressive, muscle-wasting disease, characterized by progressive deterioration of skeletal muscle that causes rapid loss of mobility. The failure in respiratory and cardiac muscles is the underlying cause of premature death in most patients with DMD. Mut...
上市的9种ASO药物中有8种是用于治疗罕见病,分别用于治疗Duchenne型肌营养不良症(Duchenne muscular dystrophy,DMD)(4种)、纯合子家族性高胆固醇血症(1种)、家族性淀粉样多发神经病变(1种)、脊髓性肌萎缩症(1种)和家族性乳糜微粒血症...
Duchenne muscular dystrophy: an overview Duchenne muscular dystrophy (DMD, OMIM#310200) is a progressive, incurable, X-linked genetic disease that affects 1 in 5000–6000 boys. The disease is caused by the lack of functional dystrophin, due to over 7000 patient-specific mutations inDMD, one of...