Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)

McGovern Margaret M.; Wasserstein Melissa P.; Kirmse Brian; Duvall W. Lane; Schiano Thomas; Thurberg Beth L.; Richards Susan; Cox Gerald F.

首页> 外文期刊>Genetics in medicine >Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)

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Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)

机译：在患有Niemann-Pick病B型（酸性鞘磷脂酶缺乏症）的成年人中进行olipudase alfa（重组人酸性鞘磷脂酶）I期试验的新型第一剂药物不良反应

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Purpose: Enzyme replacement therapy with olipudase alfa (recombinant human acid sphingomyelinase) is being developed for Niemann-Pick disease type B (NPD B).

机译：目的：正在开发针对尼曼-匹克病B型（NPD B）的用脂酶Alfa（重组人酸性鞘磷脂酶）进行酶替代疗法。

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《Genetics in medicine 》 |2016年第1期| 共7页
作者
McGovern Margaret M.; Wasserstein Melissa P.; Kirmse Brian; Duvall W. Lane; Schiano Thomas; Thurberg Beth L.; Richards Susan; Cox Gerald F.;
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正文语种 eng
中图分类医学遗传学 ;
关键词
acute phase reaction; ceramide; hyperbilirubinemia; Niemann-Pick disease type B; recombinant human acid sphingomyelinase;

机译：急性期反应神经酰胺高胆红素血症尼曼-匹克病B型重组人酸性鞘磷脂酶;

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1. Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency) [J] . McGovern Margaret M., Wasserstein Melissa P., Kirmse Brian, Genetics in medicine . 2016 ,第1期

机译：在患有Niemann-Pick病B型（酸性鞘磷脂酶缺乏症）的成年人中进行olipudase alfa（重组人酸性鞘磷脂酶）I期试验的新型第一剂药物不良反应
2. Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months [J] . Melissa P. Wasserstein, George A. Diaz, Robin H. Lachmann, Journal of inherited metabolic disease . 2018 ,第5期

机译：用于治疗酸鞘氨基酶缺乏（ASMD）的橄榄酶ALFA：治疗30个月的成人的安全性和疗效
3. Olipudase alfa for the treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months [J] . Puga Ma Cristina, Wasserstein Melissa, Diaz George, Molecular genetics and metabolism . 2017 ,第1a2期

机译：橄榄酶Alfa用于治疗酸性鞘氨键酶缺乏（ASMD）：治疗30个月的成人的安全性和有效性
4. The reproductive pathology of acid sphingomyelinase deficient mice and development of methods to prevent Niemann-Pick disease. [D] . Butler, Andrew Avigdor. 2002

机译：酸性鞘磷脂酶缺陷型小鼠的生殖病理学和预防尼曼-皮克病的方法的发展。
5. Liver and Skin Histopathology in Adults with Acid Sphingomyelinase Deficiency (Niemann-Pick Disease Type B) [O] . Beth L. Thurberg, Melissa P. Wasserstein, Thomas Schiano, -1

机译：在酸性鞘磷脂酶缺乏症成人肝脏和皮肤组织病理学（尼曼匹克病B型）
6. Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency) [O] . Margaret M. McGovern, Melissa P. Wasserstein, Brian Kirmse, 2015

机译：新型第一剂量不良药物在具有Niemann-pick疾病B（酸鞘氨基氨基酶缺乏）的成人中的橄榄酶Alfa（重组人酸鞘氨基氨基酶）试验期间
7. Relationships Among Cerebrospinal Fluid Human Immunodeficiency Virus Type 1 (HIV-1) Culture, Quinolinic Acid Level, and Reaction Time in HIV-1-Infected Individuals [R] . Mapou, R. L., Law, W. A., Heyes, M. P., 1995

机译：HIV-1感染个体中脑脊液人类免疫缺陷病毒1型（HIV-1）培养，喹啉酸水平和反应时间的关系

Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency)

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