中国实用神经疾病杂志2024，Vol.27Issue(1)：119-123,5.DOI:10.12083/SYSJ.230935
伐莫洛酮治疗Duchenne型肌营养不良症的研究进展
Research progress of vamorolone in the treatment of Duchenne muscular dystrophy
摘要
Abstract
Duchenne muscular dystrophy(DMD),which is the most common type of progressive muscular dystrophy(PMD),is also the most common X-linked recessive myopathy in China.The incidence rate of about 30/100 000 male infants,the main performances of DMD are slowly aggravated trunk muscles,limb muscles weakness and atrophy,false hypertrophy of the muscles,most will be accompanied by myocardial damage.General children at 12 years old can not walk,later will appear respiratory muscle weakness.Because of no characteristic treatment,most patients with DMD in 20-30 years old will be death due to respiratory tract infection and heart failure.Many studies have found that glucocorticoids can significantly improve motor and cardiac function in DMD.Currently,early use of glucocorticoids is recommended in clinic,but clinical application of glucocorticoids is more limited because of the adverse reactions.In recent 10 years,foreign clinical studies have found that vamorolone has the same therapeutic effect as glucocorticoid in the treatment of DMD,but lack of the adverse effects associated with glucocorticoid(growth retarding,bone disease,muscle atrophy).This article reviews the mechanism,treatment and side effects of vamorolone in the treatment of DMD.关键词
Duchenne型肌营养不良症/伐莫洛酮/糖皮质激素/新型抗炎药游离甾体化合物Key words
Duchenne muscular dystrophy/Vamorolone/Glucocorticoid/Novel anti-inflammatory free steroid compounds分类
临床医学引用本文复制引用
王琴,焦琳娜,何建丽,宋志彬..伐莫洛酮治疗Duchenne型肌营养不良症的研究进展[J].中国实用神经疾病杂志,2024,27(1):119-123,5.基金项目
中山市医学科研项目(编号:2021A020318) （编号:2021A020318）
