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Correction of a short cardiac PR interval in a 12-year-old girl with late-onset Pompe disease following enzyme replacement therapy.

作者信息

Fernández Cristina, Legido Agustín, Jethva Reena, Marks Harold G

出版信息

Genet Med. 2012 Aug;14(8):757-8. doi: 10.1038/gim.2012.61.

DOI:10.1038/gim.2012.61
PMID:22863876
Abstract
摘要

相似文献

1
Correction of a short cardiac PR interval in a 12-year-old girl with late-onset Pompe disease following enzyme replacement therapy.酶替代疗法后一名12岁晚发型庞贝病女孩心脏PR间期缩短的纠正
Genet Med. 2012 Aug;14(8):757-8. doi: 10.1038/gim.2012.61.
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引用本文的文献

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What's new and what's next for gene therapy in Pompe disease?庞贝病基因治疗的新进展和未来方向是什么?
Expert Opin Biol Ther. 2022 Sep;22(9):1117-1135. doi: 10.1080/14712598.2022.2067476. Epub 2022 Apr 27.
2
Advancements in AAV-mediated Gene Therapy for Pompe Disease.腺相关病毒介导的庞贝病基因治疗的进展。
J Neuromuscul Dis. 2020;7(1):15-31. doi: 10.3233/JND-190426.
3
Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.
腺相关病毒介导的α-葡萄糖苷酶(rAAV1-CMV-hGAA)基因疗法经膈给药治疗庞贝病患儿的安全性
Hum Gene Ther Clin Dev. 2017 Dec;28(4):208-218. doi: 10.1089/humc.2017.146.
4
Therapeutic advances in the management of Pompe disease and other metabolic myopathies.庞贝病和其他代谢性肌病的治疗进展。
Ther Adv Neurol Disord. 2013 Sep;6(5):311-21. doi: 10.1177/1756285613487570.