Enzyme replacement therapy in India: Lessons and insights.
作者信息
Muranjan M, Karande S
机构信息
Department of Pediatrics, Seth GS Medical College and KEM Hospital, Parel, Mumbai, Maharashtra, India.
出版信息
J Postgrad Med. 2018 Oct-Dec;64(4):195-199. doi: 10.4103/jpgm.JPGM_41_18.
Abstract
摘要
相似文献
1
Enzyme replacement therapy in India: Lessons and insights.印度的酶替代疗法：经验与见解。
J Postgrad Med. 2018 Oct-Dec;64(4):195-199. doi: 10.4103/jpgm.JPGM_41_18.
2
Eighth International Symposium on Lysosomal Storage Diseases.
Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S1-3.
3
Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges.溶酶体贮积症的酶替代治疗：20 年经验教训及尚存挑战
Annu Rev Genomics Hum Genet. 2012;13:307-35. doi: 10.1146/annurev-genom-090711-163739.
4
Recent advances in treatment approaches of lysosomal storage diseases (LSDs).
Curr Pharm Biotechnol. 2011 Jun;12(6):853. doi: 10.2174/138920111795542660.
5
Novel therapies and future perspectives.新型疗法与未来展望。
Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S109-10. doi: 10.5414/cpp47109.
6
Diagnosis and management of infusion-related hypersensitivity reactions to enzyme replacement therapy for lysosomal diseases: The role of desensitization.
J Allergy Clin Immunol Pract. 2016 Mar-Apr;4(2):354-6. doi: 10.1016/j.jaip.2015.11.012. Epub 2016 Jan 5.
7
Current state of the management of LSDs.溶酶体贮积症的当前管理状况。
Int J Clin Pharmacol Ther. 2009;47 Suppl 1:S61-2.
8
ERT Degrades Gene Therapy for Storage Disorder.ERT可降解用于贮积症的基因疗法。
Mol Ther. 2019 Jul 3;27(7):1207-1208. doi: 10.1016/j.ymthe.2019.06.002. Epub 2019 Jun 13.
9
Therapy for lysosomal storage disorders.溶酶体贮积症的治疗。
IUBMB Life. 2010 Jan;62(1):33-40. doi: 10.1002/iub.284.
10
Lysosomal storage disorders: 4 big questions.溶酶体贮积症：4个重大问题。
Nature. 2016 Sep 22;537(7621):S165. doi: 10.1038/537S165a.
引用本文的文献
1
Plant molecular farming: a promising frontier for orphan drug production.植物分子农业：孤儿药生产的一个有前景的前沿领域。
Biotechnol Lett. 2025 May 17;47(3):56. doi: 10.1007/s10529-025-03596-2.
2
Economic Burden of Gaucher Disease at a Tertiary Care Public Hospital in Mumbai.孟买一家三级公立医院戈谢病的经济负担。
Indian J Pediatr. 2024 May;91(5):463-469. doi: 10.1007/s12098-023-04740-4. Epub 2023 Jul 24.
3
Lysosomal storage disorders: from biology to the clinic with reference to India.溶酶体贮积症：结合印度情况从生物学进展到临床应用
Lancet Reg Health Southeast Asia. 2022 Nov 21;9:100108. doi: 10.1016/j.lansea.2022.100108. eCollection 2023 Feb.
4
Advances in orphan drug development: Time to change the status and stereotype.罕见病药物研发进展：是时候改变现状与刻板印象了。
Perspect Clin Res. 2022 Jul-Sep;13(3):123-128. doi: 10.4103/picr.picr_193_21. Epub 2022 Jun 30.
5
Transformative effect of a Humanitarian Program for individuals affected by rare diseases: building support systems and creating local expertise.人道主义计划对罕见病患者的变革性影响：构建支持系统和培养本土专业知识。
Orphanet J Rare Dis. 2022 Apr 4;17(1):87. doi: 10.1186/s13023-022-02192-1.
6
Challenges in Chronic Genetic Disorders: Lessons From the COVID-19 Pandemic.慢性遗传疾病面临的挑战：新冠疫情带来的启示。
Indian Pediatr. 2021 Apr 15;58(4):391-392. doi: 10.1007/s13312-021-2202-x.
7
Spectrum of Lysosomal Storage Disorders at Tertiary Centre: Retrospective Case-Record Analysis.三级医疗中心溶酶体贮积症的谱系：回顾性病例记录分析
J Pediatr Genet. 2020 Jun;9(2):87-92. doi: 10.1055/s-0039-3402070. Epub 2020 Jan 2.
8
Genomics of rare genetic diseases-experiences from India.罕见遗传病的基因组学：来自印度的经验。
Hum Genomics. 2019 Sep 25;14(1):52. doi: 10.1186/s40246-019-0215-5.
9
The role of patient organizations in the rare disease ecosystem in India: an interview based study.患者组织在印度罕见病生态系统中的作用：基于访谈的研究。
Orphanet J Rare Dis. 2019 May 29;14(1):117. doi: 10.1186/s13023-019-1093-6.
本文引用的文献
1
2
State of rare disease management in Southeast Asia.东南亚罕见病管理状况。
Orphanet J Rare Dis. 2016 Aug 2;11(1):107. doi: 10.1186/s13023-016-0460-9.
3
Outcome of Gaucher Disease in India: Lessons from Prevalent Diagnostic and Therapeutic Practices.
Indian Pediatr. 2016 Aug 8;53(8):685-8. doi: 10.1007/s13312-016-0910-4. Epub 2016 Jun 1.
4
Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials.维拉苷酶α（VPRIV）用于戈谢病患者的酶替代疗法：III期临床试验的长期数据。
Am J Hematol. 2015 Jul;90(7):584-91. doi: 10.1002/ajh.24012.
5
Enzyme replacement therapy for lysosomal diseases: lessons from 20 years of experience and remaining challenges.溶酶体贮积症的酶替代治疗：20 年经验教训及尚存挑战
Annu Rev Genomics Hum Genet. 2012;13:307-35. doi: 10.1146/annurev-genom-090711-163739.
6
Predicting cross-reactive immunological material (CRIM) status in Pompe disease using GAA mutations: lessons learned from 10 years of clinical laboratory testing experience.利用 GAA 突变预测庞贝病中的交叉反应性免疫物质（CRIM）状态：来自 10 年临床实验室检测经验的教训。
Am J Med Genet C Semin Med Genet. 2012 Feb 15;160C(1):40-9. doi: 10.1002/ajmg.c.31319. Epub 2012 Jan 17.
7
Recombinant macrophage targeted enzyme replacement therapy for Gaucher disease in India.印度的巨噬细胞靶向重组酶替代疗法治疗戈谢病。
Indian Pediatr. 2011 Oct;48(10):779-84. doi: 10.1007/s13312-011-0128-4.
8
New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy.溶酶体贮积症的新治疗选择：酶替代疗法、小分子疗法和基因疗法。
Hum Genet. 2007 Mar;121(1):1-22. doi: 10.1007/s00439-006-0280-4. Epub 2006 Nov 7.
9
Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products.罕见病仍是无人问津的孤儿还是已被欣然接纳？开发和使用孤儿药面临的挑战。
Br J Clin Pharmacol. 2006 Sep;62(3):264-71. doi: 10.1111/j.1365-2125.2006.02654.x.
10
Pompe disease diagnosis and management guideline.庞贝病诊断与管理指南。
Genet Med. 2006 May;8(5):267-88. doi: 10.1097/01.gim.0000218152.87434.f3.
Zotero 插件