做好准备迎接Duchenne型肌营养不良症基因治疗时代的到来
做好准备迎接Duchenne型肌营养不良症基因治疗时代的到来
Publication information
Publisher
中山大学附属第一医院神经科,广州,510080
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Scope and Contents
Contents
近年来国外关于Duchenne型肌营养不良症基因治疗的临床试验取得了一定成果,疗效和安全性良好.我国Duchenne型肌营养不良症病例数较多,目前已纳入首批罕见病目录,可以预测基因治疗将很快进入我国.临床医师、患儿及其家属、政府相关部门、慈善基金会、媒体均应做好相应的准备,这将使我国Duchenne型肌营养不良症患儿获益.
Alternative Titles
Full title
做好准备迎接Duchenne型肌营养不良症基因治疗时代的到来
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Author / Creator
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Primary Identifiers
Record Identifier
TN_cdi_wanfang_journals_xdsjjbzz201905002
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_wanfang_journals_xdsjjbzz201905002
Other Identifiers
ISSN
1672-6731
DOI
10.3969/j.issn.1672-6731.2019.05.002
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