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Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.

作者信息

Mistry Pramod K, Balwani Manisha, Baris Hagit N, Turkia Hadhami Ben, Burrow T Andrew, Charrow Joel, Cox Gerald F, Danda Sumita, Dragosky Marta, Drelichman Guillermo, El-Beshlawy Amal, Fraga Cristina, Freisens Selena, Gaemers Sebastiaan, Hadjiev Evgueniy, Kishnani Priya S, Lukina Elena, Maison-Blanche Pierre, Martins Ana Maria, Pastores Gregory, Petakov Milan, Peterschmitt M Judith, Rosenbaum Hanna, Rosenbloom Barry, Underhill Lisa H, Cox Timothy M

机构信息

Yale University School of Medicine, New Haven, CT, USA.

Icahn School of Medicine at Mount Sinai, New York, NY, USA.

出版信息

Blood Cells Mol Dis. 2018 Jul;71:71-74. doi: 10.1016/j.bcmd.2018.04.001. Epub 2018 Apr 9.

DOI:10.1016/j.bcmd.2018.04.001
PMID:29680197
Abstract
摘要

相似文献

1
Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.作为1型戈谢病一线治疗药物的 eliglustat 的安全性、有效性及获批情况。
Blood Cells Mol Dis. 2018 Jul;71:71-74. doi: 10.1016/j.bcmd.2018.04.001. Epub 2018 Apr 9.
2
Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States.美国关于使用依利格鲁司他治疗1型戈谢病成人患者的建议。
Mol Genet Metab. 2016 Feb;117(2):95-103. doi: 10.1016/j.ymgme.2015.09.002. Epub 2015 Sep 7.
3
Should eliglustat be first line therapy for patients with type 1 Gaucher disease? Definitions of safety and efficacy.eliglustat是否应为1型戈谢病患者的一线治疗方法？安全性和有效性的定义。
Blood Cells Mol Dis. 2018 Feb;68:14-16. doi: 10.1016/j.bcmd.2017.09.003. Epub 2017 Sep 14.
4
Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.欧洲1型戈谢病成年患者使用依利格鲁司他的管理和监测建议
Eur J Intern Med. 2017 Jan;37:25-32. doi: 10.1016/j.ejim.2016.07.011. Epub 2016 Aug 10.
5
Addendum to Letter to the Editor: Safety, efficacy, and authorization of eliglustat as a first-line therapy in Gaucher disease type 1.致编辑信的附录：艾考糊精作为1型戈谢病一线治疗药物的安全性、有效性及获批情况
Blood Cells Mol Dis. 2019 Jul;77:101-102. doi: 10.1016/j.bcmd.2019.04.003. Epub 2019 Apr 9.
6
Outcomes after 18 months of eliglustat therapy in treatment-naïve adults with Gaucher disease type 1: The phase 3 ENGAGE trial.18个月依利格鲁司他治疗初治1型戈谢病成年患者的疗效：3期ENGAGE试验。
Am J Hematol. 2017 Nov;92(11):1170-1176. doi: 10.1002/ajh.24877. Epub 2017 Oct 3.
7
Case report of unexpected gastrointestinal involvement in type 1 Gaucher disease: comparison of eliglustat tartrate treatment and enzyme replacement therapy.1型戈谢病意外累及胃肠道的病例报告：酒石酸 eliglustat 治疗与酶替代疗法的比较
BMC Med Genet. 2017 May 15;18(1):55. doi: 10.1186/s12881-017-0403-x.
8
A pooled analysis of adverse events in 393 adults with Gaucher disease type 1 from four clinical trials of oral eliglustat: Evaluation of frequency, timing, and duration.一项对来自四项口服依利格鲁司他临床试验的393例1型戈谢病成人患者不良事件的汇总分析：频率、时间和持续时间评估。
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Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients.1型戈谢病患者对底物还原疗法与酶替代疗法的生化反应。
Orphanet J Rare Dis. 2016 Mar 24;11:28. doi: 10.1186/s13023-016-0413-3.
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Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.来自四项已完成的口服 eliglustat 治疗 1 型戈谢病成人患者的长期不良事件概况。
Orphanet J Rare Dis. 2019 Jun 7;14(1):128. doi: 10.1186/s13023-019-1085-6.

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