{"title":"结节性硬化症的治疗进展。","authors":"L P Zou","doi":"10.3760/cma.j.cn112140-20230404-00238","DOIUrl":null,"url":null,"abstract":"结节性硬化症是一种常染色体显性遗传的神经皮肤综合征，可累及全身系统，特别是脑、皮肤、神经系统、肾等多器官受累。随着对其致病机制的深入研究，针对病因的靶向药物哺乳动物雷帕霉素靶蛋白抑制剂逐步应用于临床，国内外临床研究证明可有效改善其临床症状。.","PeriodicalId":23998,"journal":{"name":"Zhonghua er ke za zhi = Chinese journal of pediatrics","volume":null,"pages":null},"PeriodicalIF":0.0000,"publicationDate":"2023-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"[Progress in the treatment of tuberous sclerosis complex].\",\"authors\":\"L P Zou\",\"doi\":\"10.3760/cma.j.cn112140-20230404-00238\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"结节性硬化症是一种常染色体显性遗传的神经皮肤综合征，可累及全身系统，特别是脑、皮肤、神经系统、肾等多器官受累。随着对其致病机制的深入研究，针对病因的靶向药物哺乳动物雷帕霉素靶蛋白抑制剂逐步应用于临床，国内外临床研究证明可有效改善其临床症状。.\",\"PeriodicalId\":23998,\"journal\":{\"name\":\"Zhonghua er ke za zhi = Chinese journal of pediatrics\",\"volume\":null,\"pages\":null},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2023-08-02\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Zhonghua er ke za zhi = Chinese journal of pediatrics\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.3760/cma.j.cn112140-20230404-00238\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Zhonghua er ke za zhi = Chinese journal of pediatrics","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.3760/cma.j.cn112140-20230404-00238","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
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