杜氏肌营养不良
肌营养不良蛋白
肌营养不良
肌肉无力
弱点
变性(医学)
生物信息学
医学
病理
生物
外科
内科学
标识
DOI:10.1016/s1474-4422(24)00082-6
摘要
Duchenne muscular dystrophy is a lethal X-linked genetic disorder marked by progressive muscle weakness and degeneration. Mutations in the dystrophin gene lead to an absence of the dystrophin protein, which is essential for muscle cell integrity, resulting in muscle damage and decreased mobility. In addition to treating symptoms, corticosteroids have been used for the past three decades to slow the progression of disease.1 However, despite the benefits of corticosteroids in delaying loss of ambulation and enhancing quality of life, their long-term use is associated with considerable side effects, including weight gain and bone fragility.
科研通智能强力驱动
Strongly Powered by AbleSci AI