Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
Standard of care versus new-wave corticosteroids in the treatment of Duchenne muscular dystrophy: Can we do better?
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DOI：
10.1186/s13023-021-01758-9
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发表时间：
2021-03-04
影响因子：
3.7
通讯作者：
Rybalka E
中科院分区：
文献类型：
--
作者：
Kourakis S;Timpani CA;Campelj DG;Hafner P;Gueven N;Fischer D;Rybalka E
药物皮质类固醇治疗是杜氏肌营养不良症（DMD）的标准治疗，旨在通过强效抗炎作用控制症状和减缓疾病进展。然而，一个主要的问题是与长期使用相关的显著不良反应。本综述讨论了DMD标准治疗的利弊，并将其与新一代游离皮质类固醇-实验抗炎药物的现状也进行了审查，与见解替代药物，可以提供治疗优势。虽然新型游离类固醇可能是皮质类固醇的上级替代品，但应探索其他潜在的治疗方法。除了抗炎之外，重新利用或开发能够解决DMD的许多致病特征的新型药理学疗法可以产生更大的治疗优势。
Pharmacological corticosteroid therapy is the standard of care in Duchenne Muscular Dystrophy (DMD) that aims to control symptoms and slow disease progression through potent anti-inflammatory action. However, a major concern is the significant adverse effects associated with long term-use. This review discusses the pros and cons of standard of care treatment for DMD and compares it to novel data generated with the new-wave dissociative corticosteroid, vamorolone. The current status of experimental anti-inflammatory pharmaceuticals is also reviewed, with insights regarding alternative drugs that could provide therapeutic advantage. Although novel dissociative steroids may be superior substitutes to corticosteroids, other potential therapeutics should be explored. Repurposing or developing novel pharmacological therapies capable of addressing the many pathogenic features of DMD in addition to anti-inflammation could elicit greater therapeutic advantages.
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影响因子：
4
作者：
Creelan BC;Gabrilovich DI;Gray JE;Williams CC;Tanvetyanon T;Haura EB;Weber JS;Gibney GT;Markowitz J;Proksch JW;Reisman SA;McKee MD;Chin MP;Meyer CJ;Antonia SJ
通讯作者：
Antonia SJ
影响因子：
4.1
作者：
Coutinho, Agnes E.;Chapman, Karen E.
通讯作者：
Chapman, Karen E.
影响因子：
3.4
作者：
ANGELINI, C;PEGORARO, E;COSTA, C
通讯作者：
COSTA, C
影响因子：
5.1
作者：
Barber, Brent J.;Andrews, Jennifer G.;Cunniff, Christopher
通讯作者：
Cunniff, Christopher
影响因子：
5.8
作者：
Breuer, Johanna;Herich, Sebastian;Schwab, Nicholas
通讯作者：
Schwab, Nicholas