Institute for Health and Sport (IHeS), Victoria University, Melbourne, VIC, Australia.
Australian Institute for Musculoskeletal Science (AIMSS), St Albans, VIC, Australia.
Orphanet J Rare Dis. 2021 Mar 4;16(1):117. doi: 10.1186/s13023-021-01758-9.
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. MAIN: 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.
在杜氏肌营养不良症（DMD）中，药物性皮质类固醇治疗是标准的治疗方法，旨在通过强效的抗炎作用来控制症状和减缓疾病进展。然而，人们主要关注的是长期使用所带来的显著不良反应。
本综述讨论了 DMD 标准治疗的利弊，并将其与新型分离型皮质类固醇药物沃莫罗龙的新数据进行了比较。还回顾了抗炎性药物的当前研究状况，探讨了可能具有治疗优势的替代药物。
虽然新型分离型类固醇可能是皮质类固醇的更好替代品，但还应探索其他潜在的治疗方法。除了抗炎作用之外，重新利用或开发新的药理学疗法来解决 DMD 的多种致病特征，可能会产生更大的治疗优势。
