Olipudase alfa approved for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG).
作者信息
Penon-Portmann Monica, Poskanzer Sheri A, Ganesh Jaya, Chang Irene
机构信息
Division of Genetic Medicine, Department of Pediatrics, University of Washington, Seattle, WA.
St. Luke's Health System, Boise, ID.
出版信息
Genet Med Open. 2023 Apr 17;1(1):100780. doi: 10.1016/j.gimo.2023.100780. eCollection 2023.
Abstract
摘要
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Olipudase Alfa: First Approval.阿糖苷酶α：首次获批
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A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results.一项评估奥利普酶阿尔法酶替代疗法治疗成人慢性酸性鞘磷脂酶缺乏症（ASMD）的随机、安慰剂对照临床试验：一年的结果。
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