Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy
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چکیده
منابع مشابه
Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy
Follistatin is a ubiquitous secretory propeptide that functions as a potent inhibitor of the myostatin pathway, resulting in an increase in skeletal muscle mass. Its ability to interact with the pituitary activin-inhibin axis and suppress the secretion of follicle-stimulating hormone (FSH) called for caution in its clinical applicability. This limitation was circumvented by the use of one of th...
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University of Medicine and Dentistry of New Jersey, 401, Haddon Avenue Camden, New Jersey, USA e-mail: fayssoil2000@ yahoo.fr
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Valley sign has been described in patients with Duchenne muscular dystrophy (DMD). As there are genetic and clinical similarities between DMD and Becker muscular dystrophy (BMD), this clinical sign is evaluated in this study in BMD and DMD/BMD outliers. To evaluate the sign, 28 patients with Becker muscular dystrophy (BMD), 8 DMD/BMD outliers and 44 age-matched male controls with other neuromus...
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A benign Becker muscular dystrophy (BMD) patient with a marked decrease in dystrophin exhibited remarkable expression of dystrophin-related protein (DRP) on most of the muscle cell membrane. A phenotypic Duchenne muscular dystrophy patient with a truncated form of dystrophin exhibited no DRP expression on the muscle cell membrane except for the neuromuscular junction. Increased DRP expression m...
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ژورنال
عنوان ژورنال: Journal of Neuromuscular Diseases
سال: 2015
ISSN: 2214-3599,2214-3602
DOI: 10.3233/jnd-150083