• 文献检索
  • 文档翻译
  • 深度研究
  • 学术资讯
  • Suppr Zotero 插件Zotero 插件
  • 邀请有礼
  • 套餐&价格
  • 历史记录
应用&插件
Suppr Zotero 插件Zotero 插件浏览器插件Mac 客户端Windows 客户端微信小程序
定价
高级版会员购买积分包购买API积分包
服务
文献检索文档翻译深度研究API 文档MCP 服务
关于我们
关于 Suppr公司介绍联系我们用户协议隐私条款
关注我们

Suppr 超能文献

核心技术专利：CN118964589B侵权必究
粤ICP备2023148730 号-1Suppr @ 2026

文献检索

告别复杂PubMed语法，用中文像聊天一样搜索，搜遍4000万医学文献。AI智能推荐，让科研检索更轻松。

立即免费搜索

文件翻译

保留排版，准确专业，支持PDF/Word/PPT等文件格式，支持 12+语言互译。

免费翻译文档

深度研究

AI帮你快速写综述，25分钟生成高质量综述，智能提取关键信息，辅助科研写作。

立即免费体验

1型戈谢病的两种底物还原疗法并不等效。对休斯等人的评论。戈谢病患者在酶替代疗法和底物还原疗法之间的转换：来自戈谢病结果调查（GOS）的数据。2022年，，5158。

The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). 2022, , 5158.

作者信息

Mistry Pramod K, Kishnani Priya S, Balwani Manisha, Charrow Joel M, Hull Judy, Weinreb Neal J, Cox Timothy M

机构信息

Department of Medicine, Pediatrics, and Cellular & Molecular Physiology, Yale University School of Medicine, 20 York Street, New Haven, CT 06510, USA.

Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

出版信息

J Clin Med. 2023 May 4;12(9):3269. doi: 10.3390/jcm12093269.

DOI:10.3390/jcm12093269
PMID:37176709
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10179580/
Abstract

In their paper, Hughes et al. [...].

摘要

在他们的论文中，休斯等人……

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/79f8/10179580/3d11ce8bdde0/jcm-12-03269-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/79f8/10179580/3d11ce8bdde0/jcm-12-03269-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/79f8/10179580/3d11ce8bdde0/jcm-12-03269-g001.jpg

相似文献

1
The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). 2022, , 5158.1型戈谢病的两种底物还原疗法并不等效。对休斯等人的评论。戈谢病患者在酶替代疗法和底物还原疗法之间的转换：来自戈谢病结果调查（GOS）的数据。2022年，，5158。
J Clin Med. 2023 May 4;12(9):3269. doi: 10.3390/jcm12093269.
2
Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). 2022, , 5158".对米斯特里等人的回复。1型戈谢病的两种底物还原疗法并不等效。对“休斯等人。戈谢病患者酶替代疗法和底物还原疗法之间的转换：来自戈谢病结局调查（GOS）的数据。2022年，，5158”的评论。
J Clin Med. 2023 Jun 13;12(12):4017. doi: 10.3390/jcm12124017.
3
Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).戈谢病患者在酶替代疗法和底物减少疗法之间的转换：来自戈谢病结果调查（GOS）的数据。
J Clin Med. 2022 Aug 31;11(17):5158. doi: 10.3390/jcm11175158.
4
Enzyme replacement and substrate reduction therapy for Gaucher disease.戈谢病的酶替代疗法和底物减少疗法。
Cochrane Database Syst Rev. 2015 Mar 27;2015(3):CD010324. doi: 10.1002/14651858.CD010324.pub2.
5
Characteristics of 26 patients with type 3 Gaucher disease: A descriptive analysis from the Gaucher Outcome Survey.26例3型戈谢病患者的特征：来自戈谢病结局调查的描述性分析
Mol Genet Metab Rep. 2017 Dec 27;14:73-79. doi: 10.1016/j.ymgmr.2017.10.011. eCollection 2018 Mar.
6
Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports.两例1型戈谢病患者联合使用米格鲁司他和酶替代疗法：两例病例报告
J Med Case Rep. 2018 Jan 27;12(1):19. doi: 10.1186/s13256-017-1541-7.
7
Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report.一名成年1型戈谢病患者成功从酶替代疗法转换为米格鲁司他治疗：病例报告
J Med Case Rep. 2016 Nov 8;10(1):315. doi: 10.1186/s13256-016-1060-y.
8
Treatment patterns from 647 patients with Gaucher disease: An analysis from the Gaucher Outcome Survey.647例戈谢病患者的治疗模式：来自戈谢病结局调查的分析。
Blood Cells Mol Dis. 2018 Feb;68:218-225. doi: 10.1016/j.bcmd.2016.10.014. Epub 2016 Oct 20.
9
Enzyme Replacement or Substrate Reduction? A Review of Gaucher Disease Treatment Options.酶替代疗法还是底物减少疗法？戈谢病治疗方案综述。
Hosp Pharm. 2016 Jul;51(7):553-63. doi: 10.1310/hpj5107-553.
10
Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.将伊米苷酶换用米格鲁司他治疗法国1型戈谢病患者：病例系列
J Med Case Rep. 2015 Jun 23;9:146. doi: 10.1186/s13256-015-0617-5.

引用本文的文献

1
Eliglustat and cardiac comorbidities in Gaucher disease: a pharmacogenomic approach to safety and efficacy.依利格鲁司他与戈谢病的心脏合并症：一种关于安全性和疗效的药物基因组学方法
Front Med (Lausanne). 2025 Mar 17;12:1535099. doi: 10.3389/fmed.2025.1535099. eCollection 2025.
2
Exploration of phytoconstituents of Medhya Rasayana herbs to identify potential inhibitors for through high-throughput screening.探索益智药草的植物成分，通过高通量筛选确定潜在抑制剂。
Front Mol Biosci. 2024 Oct 9;11:1476482. doi: 10.3389/fmolb.2024.1476482. eCollection 2024.
3
Screening of phytoconstituents from Bacopa monnieri (L.) Pennell and Mucuna pruriens (L.) DC. to identify potential inhibitors against Cerebroside sulfotransferase.

本文引用的文献

1
Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS).戈谢病患者在酶替代疗法和底物减少疗法之间的转换：来自戈谢病结果调查（GOS）的数据。
J Clin Med. 2022 Aug 31;11(17):5158. doi: 10.3390/jcm11175158.
2
How a concentration-effect analysis of data from the eliglustat thorough electrocardiographic study was used to support dosing recommendations.如何利用依利格鲁司他全面心电图研究的数据浓度-效应分析来支持剂量建议。
Mol Genet Metab. 2020 Sep-Oct;131(1-2):211-218. doi: 10.1016/j.ymgme.2020.09.003. Epub 2020 Sep 16.
3
从水苏糖（Bacopa monnieri (L.) Pennell）和黎豆（Mucuna pruriens (L.) DC.）中筛选植物成分，以鉴定对神经酰胺硫酸转移酶有潜在抑制作用的物质。
PLoS One. 2024 Oct 24;19(10):e0307374. doi: 10.1371/journal.pone.0307374. eCollection 2024.
4
Phytoconstituents of Withania somnifera (L.) Dunal (Ashwagandha) unveiled potential cerebroside sulfotransferase inhibitors: insight through virtual screening, molecular dynamics, toxicity, and reverse pharmacophore analysis.睡茄（Withania somnifera (L.) Dunal，印度人参）的植物成分揭示了潜在的脑苷脂磺基转移酶抑制剂：通过虚拟筛选、分子动力学、毒性和反向药效团分析获得的见解
J Biol Eng. 2024 Oct 23;18(1):59. doi: 10.1186/s13036-024-00456-x.
5
Mysterious sphingolipids: metabolic interrelationships at the center of pathophysiology.神秘的鞘脂类：病理生理学核心的代谢相互关系
Front Physiol. 2024 Jan 3;14:1229108. doi: 10.3389/fphys.2023.1229108. eCollection 2023.
6
Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). 2022, , 5158".对米斯特里等人的回复。1型戈谢病的两种底物还原疗法并不等效。对“休斯等人。戈谢病患者酶替代疗法和底物还原疗法之间的转换：来自戈谢病结局调查（GOS）的数据。2022年，，5158”的评论。
J Clin Med. 2023 Jun 13;12(12):4017. doi: 10.3390/jcm12124017.
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.
4
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.
5
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。
Blood Cells Mol Dis. 2018 Feb;68:185-191. doi: 10.1016/j.bcmd.2017.01.006. Epub 2017 Jan 13.
6
Eliglustat compared with imiglucerase in patients with Gaucher's disease type 1 stabilised on enzyme replacement therapy: a phase 3, randomised, open-label, non-inferiority trial.依利格鲁司他与伊米苷酶治疗稳定的 1 型戈谢病患者的比较：一项 3 期、随机、开放标签、非劣效性试验。
Lancet. 2015 Jun 13;385(9985):2355-62. doi: 10.1016/S0140-6736(14)61841-9. Epub 2015 Mar 26.
7
Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study.评估米格列醇作为稳定型 1 型戈谢病成人在酶治疗后的维持治疗：一项前瞻性、开放标签、非劣效性研究。
Orphanet J Rare Dis. 2012 Dec 27;7:102. doi: 10.1186/1750-1172-7-102.
8
ELIGLUSTAT TARTRATE: Glucosylceramide Synthase Inhibitor Treatment of Type 1 Gaucher Disease.酒石酸艾立骨化醇：葡糖神经酰胺合酶抑制剂治疗1型戈谢病。
Drugs Future. 2010 Aug 1;35(8):613-620.
9
Gastrointestinal disturbances and their management in miglustat-treated patients.接受米格列醇治疗的患者的胃肠道紊乱及其处理。
J Inherit Metab Dis. 2011 Oct;34(5):991-1001. doi: 10.1007/s10545-011-9368-7. Epub 2011 Jul 21.
10
The management of pregnancy in Gaucher disease.戈谢氏病妊娠的管理。
Eur J Obstet Gynecol Reprod Biol. 2011 May;156(1):3-8. doi: 10.1016/j.ejogrb.2010.12.024. Epub 2011 Jan 26.