ADAMTS13号
血栓性血小板减少性紫癜
重组DNA
医学
紫癜（腹足类）
儿科
免疫学
血小板
生物
基因
生态学
生物化学
化学
作者
Marie Scully,Ana G. Antun,Spero R. Cataland,Paul Coppo,Claire Dossier,Nathalie Biebuyck,Wolf-Achim Hassenpflug,Karim Kentouche,Paul Knöbl,Johanna A. Kremer Hovinga,María Fernanda López Fernández,Masanori Matsumoto,Thomas L. Ortel,Jerzy Windyga,Indranil Bhattacharya,Michael Cronin,Hong Li,Björn Mellgård,Munjal Patel,Parth Patwari
标识
DOI：10.1056/nejmoa2314793
摘要
Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived products) administered as routine prophylaxis or on-demand treatment in patients with congenital TTP is not known.
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