开展杜氏肌营养不良症更完善自然史研究的重要性。
The importance of better natural history studies for Duchenne muscular dystrophy.
作者信息
Birnkrant David J
机构信息
Case Western Reserve University School of Medicine - Pediatrics, Cleveland, Ohio, USA.
出版信息
Dev Med Child Neurol. 2025 Oct;67(10):1241-1242. doi: 10.1111/dmcn.16306. Epub 2025 Mar 18.
Abstract
This commentary is on the original article by Chesshyre et al. on pages 1280–1289 of this issue.
摘要
本评论是关于Chesshyre等人在本期第1280 - 1289页发表的原创文章。
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本文引用的文献
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Dystrophin isoform deficiency and upper-limb and respiratory function in Duchenne muscular dystrophy.杜氏肌营养不良症中的肌营养不良蛋白亚型缺乏与上肢及呼吸功能
Dev Med Child Neurol. 2025 Oct;67(10):1280-1289. doi: 10.1111/dmcn.16282. Epub 2025 Mar 14.
2
A New Perspective on Drugs for Duchenne Muscular Dystrophy: Proposals for Better Respiratory Outcomes and Improved Regulatory Pathways.杜氏肌营养不良症药物的新视角：改善呼吸结局及优化监管途径的建议
Paediatr Drugs. 2025 Mar;27(2):143-159. doi: 10.1007/s40272-024-00673-3. Epub 2024 Dec 20.
3
Cardiorespiratory management of Duchenne muscular dystrophy: emerging therapies, neuromuscular genetics, and new clinical challenges.杜氏肌营养不良的心肺管理：新兴疗法、神经肌肉遗传学和新的临床挑战。
Lancet Respir Med. 2022 Apr;10(4):403-420. doi: 10.1016/S2213-2600(21)00581-6.
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Cardiopulmonary phenotypic variability and discordance in Duchenne muscular dystrophy: Implications for new therapies.杜氏肌营养不良症的心肺表型变异性和不一致性：对新疗法的影响。
Pediatr Pulmonol. 2021 Apr;56(4):738-746. doi: 10.1002/ppul.25111. Epub 2020 Oct 22.
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Lancet Neurol. 2018 Mar;17(3):251-267. doi: 10.1016/S1474-4422(18)30024-3. Epub 2018 Feb 3.
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