关键词: agalsidase enzyme replacement therapy (ert) fabry's disease miglastat rapid review

来  源:   DOI:10.7759/cureus.37048   PDF(Pubmed)

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:
对Fabry病的介入治疗进行了综述。法布里病是一种多系统的X连锁储存障碍,影响整个身体,需要在早期进行治疗。使用“法布里病”和“管理”等关键字进行搜索,以查看数据库。从90项研究中选择了7项研究,发现米加司他和酶替代药物成功地治疗了这种疾病,而半乳糖苷酶β未能对患者产生积极影响。然而,这一分析得出了模棱两可的结论。由于分析中只包含少量研究,需要基于随机对照试验和案例研究的额外调查和评估,以确定潜在的药物相关结局.需要未来的治疗研究来治愈受遗传影响的疾病和诸如法布里病的疾病。
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