Use of advanced heart failure therapies in Duchenne muscular dystrophy
نویسندگان
چکیده
منابع مشابه
Novel therapies for Duchenne muscular dystrophy.
The development of therapeutic strategies that overcome the unique problems posed by Duchenne muscular dystrophy (DMD) has lead to the development of many contemporary approaches to human disease in general. Various treatment approaches have been explored--such as pharmacological therapies and cell-based, cytokine, and genetic therapies--that are all targeted to specific features of dystrophic ...
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Duchenne muscular dystrophy (BMD) is an inherited X-link disease. The incidence of this muscle-wasting disease is 1:5000 male live births. Mutation in the gene coding for dystrophin is the main cause of BMD. Most cases of this disease succumb to respiratory and cardiac failure in 3rd to 4th decades. The slow progression of BMD and recent achievement of gene therapies make it as an appropriate c...
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Duchenne muscular dystrophy (DMD) affects approximately 1 in 3,500 live male births [1]. It is caused by a large variety of mutations in the dystrophin gene. Because of these mutations, the body can no longer make dystrophin which is a protein important for stabilisation of the muscle cell during a contraction. Without dystrophin, muscle cells are damaged and slowly replaced by fat and scar tis...
متن کاملEmerging genetic therapies to treat Duchenne muscular dystrophy.
PURPOSE OF REVIEW Duchenne muscular dystrophy is a progressive muscle degenerative disease caused by dystrophin mutations. The purpose of this review is to highlight two emerging therapies designed to repair the primary genetic defect, called 'exon skipping' and 'nonsense codon suppression'. RECENT FINDINGS A drug, PTC124, was identified that suppresses nonsense codon translation termination....
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1 Sorbonne Université, Institut National de la Santé et de la Recherche Médicale, Association Institut de Myologie, Centre de Recherche en Myologie, UMRS974, Paris, France, 2 Laboratorio de Patologías Musculares, Fragilidad y Envejecimiento, Departamento de Ciencias Biológicas, Facultad de Ciencias Biológicas, Universidad Andres Bello, Santiago, Chile, 3 Millennium Institute on Immunology and I...
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ژورنال
عنوان ژورنال: Progress in Pediatric Cardiology
سال: 2019
ISSN: 1058-9813
DOI: 10.1016/j.ppedcard.2019.01.001