Taliglucerase Alfa in Pediatric Patients with Gaucher Disease: Efficacy, Safety, and Exploratory Gro

来源 :The 35th World Congress of the International Society of Hema | 被引量 : 0次 | 上传用户：zhengi520

【摘要】

：

【作者】

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Ari Zimran Derlis Emilio Gonzalez-Rodriguez Aya Abrahamov Deborah Elstein Alona Paz Einat Brill-Almon Raul Chertkoff Michael Wajnrajch

【机构】

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Gaucher Clinic,Shaare Zedek Medical Center and Hadassah Medical School-Hebrew University,Jerusalem,I

【出处】

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The 35th World Congress of the International Society of Hema

【发表日期】

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2014年5期

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Objective:Taliglucerase alfa is a β-glucosidase enzyme replacement therapy approved in the USA and other countries for treatment of Gaucher disease (GD) in adults,and the first approved plant cell-expressed recombinant protein.In this study,we aimed to assess the efficacy and safety of taliglucerase alfa in pediatric patients with GD.

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Taliglucerase Alfa in Pediatric Patients with Gaucher Disease: Efficacy, Safety, and Exploratory Gro

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