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法布里病及其管理:文献分析

Fabry Disease and Its Management: A Literature Analysis.

作者信息

Besekar Smruti M, Jogdand Sangita D, Naqvi Waqar M

机构信息

Pharmacology, Jawaharlal Nehru Medical College, Datta Meghe Institute of Higher Education and Research, Wardha, IND.

Dentistry, Humen Edutech, Nagpur, IND.

出版信息

Cureus. 2023 Apr 2;15(4):e37048. doi: 10.7759/cureus.37048. eCollection 2023 Apr.

Abstract

A review was conducted to evaluate interventional therapy for Fabry disease. Fabry disease is a multisystemic X-linked storage disorder that affects the entire body and needs to be treated at an early age. The search was conducted using keywords such as "Fabry disease" and "Management" to review the databases. Seven studies were chosen from the 90 studies, and it was discovered that migalastat and enzyme replacement medication were successful in treating the condition, whereas agalsidase beta failed to have a positive effect on the patient. However, this analysis produced ambiguous conclusions. As only a small number of studies were included in the analysis, additional investigations and evaluations based on randomized controlled trials and case studies are required to determine potential drug-related outcomes. There is a need for future therapeutic research to cure genetically affected illnesses and diseases such as Fabry disease.

摘要

进行了一项综述以评估法布里病的介入治疗。法布里病是一种多系统X连锁贮积病,会影响全身,需要在幼年时进行治疗。使用“法布里病”和“管理”等关键词进行检索以查阅数据库。从90项研究中选取了7项研究,发现米加司他和酶替代药物治疗该病取得了成功,而阿加糖酶β对患者未产生积极效果。然而,该分析得出的结论并不明确。由于分析中仅纳入了少量研究,因此需要基于随机对照试验和案例研究进行更多的调查和评估,以确定潜在的药物相关结果。未来需要开展治疗研究以治愈诸如法布里病等受基因影响的疾病。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/7076/10154914/a6710d9e07a0/cureus-0015-00000037048-i01.jpg

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