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典型文献
脊髓性肌萎缩症的药物治疗研究进展
文献摘要:
脊髓性肌萎缩症(SMA)是一种常染色体隐性遗传的神经元疾病,主要特征为进行性肌无力和肌肉萎缩.SMA是导致患儿在婴幼儿期死亡的主要遗传病.近年来,SMA的治疗技术及手段发展迅速,已有药物成功获批上市并应用于临床.本文就SMA的药物治疗研究进展进行综述.
文献关键词:
脊髓性肌萎缩症;运动神经元存活基因;发病机制;基因治疗;药物治疗
作者姓名:
宋语桐;张琴
作者机构:
南京医科大学附属儿童医院重症医学科,南京210008
引用格式:
[1]宋语桐;张琴-.脊髓性肌萎缩症的药物治疗研究进展)[J].中国合理用药探索,2022(05):7-10
A类:
B类:
脊髓性肌萎缩症,治疗研究,SMA,常染色体隐性遗传,肌无力,肌肉萎缩,婴幼儿期,遗传病,批上,运动神经元存活基因,基因治疗
AB值:
0.200409
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C.Hiemke;N.Bergemann;H.W.Clement;A.Conca;J.Deckert;K.Domschke;G.Eckermann;K.Egberts;M.Gerlach;C.Greiner;G.Gründer;E.Haen;U.Havemann-Reinecke;G.Hefner;R.Helmer;G.Janssen;E-.Jaquenoud;G.Laux;T.Messer;R.M?ssner;M.J.Müller;M.Paulzen;B.Pfuhlmann;P.Riederer;A.Saria;B.Schoppek;G.Schoretsanitis;M.Schwarz;M.Silva Gracia;B.Stegmann;W.Steimer;J.C.Stingl;M.Uhr;S.Ulrich;S.Unterecker;R.Waschgler;G.Zernig;G.Zurek;P.Baumann;李文标;果伟;贺静;鲍爽;路钊;牛梦溪;阮灿军;臧彦楠;王勇;张玲;刘辰庚;李洁-Department of Psychiatry and Psychotherapy,University Medical Center of Mainz,Mainz,Germany;Institute of Clinical Chemistry and Laboratory Medicine,University Medical Center of Mainz,Mainz,Germany;Kitzberg Hospitals,Center for Psychosomatic Medicine and Psychotherapy,Bad Mergentheim,Germany;Department of Child and Adolescent Psychiatry,University of Freiburg,Freiburg,Germany;Servizio Psichiatrico del Comprensorio Sanitario di Bolzano,Bolzano,Italy;Department of Psychiatry,Psychotherapy and Psychosomatics,University Hospital of Würzburg,Germany;Department of Psychiatry and Psychotherapy, Medical Center-University of Freiburg, Faculty of Medicine, University of Freiburg,Freiburg,Germany;Psychiatric Hospital,Kaufbeuren,Germany;Department of Child and Adolescent Psychiatry,Psychosomatics and Psychotherapy,Center of Mental Health,University Hospi-tal of Würzburg,Germany;Federal Institute for Drugs and Medical Devices (BfArM),Bonn,Germany;Department of Psychiatry,Psychotherapy and Psychosomatics,RWTH Aachen University,Aachen,and JARA–Translational Brain Medicine,Aachen,Germany;Clinical Pharmacology,Department of Psychiatry and Psychotherapy and Department of Pharmacology and Toxicology,Uni-versity of Regensburg,Regensburg,Germany;Department of Psychiatry and Psychosomatics,University of G?ttingen,G?ttingen,Germany;Psychiatric Hospital,Vitos Klinik,Eichberg,Eltville,Germany;Center of Epilepsy,Bielefeld,Germany;Medical Laboratory Stein,Limbach Group,M?nchengladbach,Germany;Psychiatric Hospital,K?nigsfelden,Brugg,Aargau,Switzerland;Institute of Psychological Medicine,Haag in Oberbayern,Germany;Danuviuskliniken,Psychiatric Hospital,Pfaffenhofen,Germany;Department of Psychiatry and Psychotherapy,University of Tübingen,Tübingen,Germany;Psychiatric Hospitals Oberberggruppe,Berlin,Germany;Psychiatric Hospital Weisser Hirsch,Dresden,Germany;Experimental Psychiatry Unit,Department of Psychiatry1,Medical University of Innsbruck,Innsbruck,Austria;kbo-Isar-Amper Klinikum München-Ost,Psychiatric Hospital,Munich-Haar,Germany;Department of Psychiatry,University of Bern,Bern,Switzerland;Department of Laboratory Medicine,Ludwig Maximilian University,Munich,Germany;Institute of Clinical Chemistry and Pathobiochemistry,Technical University Munich,Munich,Germany;Max Planck Institute of Psychiatry,Munich,Germany;Aristo Pharma GmbH,Berlin,Germany;Psychiatric Hospital,Feldkirch,Austria;Private Practice for Psychotherapy and Court-Certified Witness,Hall in Tirol,Austria;Medical Laboratory Bremen,Bremen,Germany;Department of Psychiatry,University of Lausanne,Prilly-Lausanne,Switzerland;中国 北京 首都医科大学附属北京安定医院,国家精神心理疾病临床医学研究中心,精神疾病诊断与治疗北京重点实验室;中国 天津 天津市安定医院
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