AAV9-mediated GAA gene therapy following enzyme replacement therapy discontinuation in children with infantile-onset Pompe disease

EClinicalMedicine 2026 Open access

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Authors: Xinting Liu, Yingying Mao, Linyan Hu, Wen He, X N Wang, Xueyuan Guo, Min Li, Yiru Wang, Yue Wang, Wenhao Ma, Yun Yuan, Zi Wang

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Shuangqing Yu, Xicheng Zhang, S S Wu, Xiaoyan Dong, Xiaobing Wu, Guang Yang

DOI: 10.1016/j.eclinm.2026.104081

Cited by: 1

References: 55

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Neurogenetic and Muscular Disorders Research

References 55