Novel Approaches to Corticoid Treatment in Duchenne Muscular Dystrophy
Novel Approaches to Corticoid Treatment in Duchenne Muscular Dystrophy
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DOI：
10.1016/j.pmr.2012.08.003
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发表时间：
2012-11-01
影响因子：
1.7
通讯作者：
Bushby, Kate
中科院分区：
文献类型：
--
作者：
Hoffman, Eric P.;Reeves, Erica;Bushby, Kate
虽然泼尼松从未被监管机构正式批准用于杜氏肌营养不良症（DMD），但其疗效已在20世纪80年代的试验中得到证实。强烈需要优化特定类型的糖皮质激素（例如，泼尼松vs地夫可特或其他）和给药方案。理想情况下，优化的方案将最大限度地提高疗效，同时最大限度地减少副作用。一项新的试验FOR-DMD旨在解决这一知识差距。与此同时，在“解离性类固醇”领域也取得了进展，这些药物能够更好地分离疗效和副作用，提供更广泛的治疗窗口。
Although prednisone has never been formally approved for use in Duchenne muscular dystrophy (DMD) by regulatory agencies, its efficacy has been confirmed in trials dating from the 1980s. There is a strong need for optimization of both specific type of glucocorticoid (eg, prednisone, vs deflazacort or others) and the dosing regimen. Ideally an optimized regimen would maximize efficacy while minimizing side-effect profiles. A new trial, FOR-DMD, aims to address this gap in knowledge. In parallel, there has been progress in the area of "dissociative steroids," drugs that are able to better separate efficacy and side effects, providing a broader therapeutic window.