Comprehensive analysis of 2097 patients with dystrophinopathy based on a database from 2011 to 2021

Abstract Background An increasing number of clinical trials for new therapeutic strategies are underway or being considered for dystrophinopathy. Having detailed data on the natural progression of this condition is crucial for assessing the effectiveness of new drugs. However, there’s a lack of data...

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Hauptverfasser:	Lei Zhao, Yiyun Shi, Chaoping Hu, Shuizhen Zhou, Hui Li, Lifeng Zhang, Chuang Qian, Yiyao Zhou, Yi Wang, Xihua Li
Format:	Artigo
Sprache:	Inglês
Veröffentlicht:	BMC 2024-08-01
Schriftenreihe:	Orphanet Journal of Rare Diseases
Schlagworte:	Dystrophinopathy Duchenne and Becker muscular dystrophies Patient management Genetic diagnosis Natural history
Online-Zugang:	https://doi.org/10.1186/s13023-024-03217-7
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