期刊
EXPERT OPINION ON BIOLOGICAL THERAPY
卷 9, 期 5, 页码 631-639出版社
INFORMA HEALTHCARE
DOI: 10.1517/14712590902902296
关键词
agalsidase alfa; enzyme replacement therapy; Fabry disease; globotriaosylceramide; pharmacodynamics
Background: Fabry disease is an X-linked lysosomal storage disease caused by deficiency of alpha-galactosidase A (alpha-Gal A), encoded by the GLA gene. The deficiency causes accumulation of neutral glycosphingolipids in various tissues, leading to neuronopathic pain, progressive renal dysfunction, cardiomyopathy and stroke. Enzyme replacement therapy (ERT) with agalsidase alfa (Replagal (TM), Shire Human Genetic Therapies) is approved for use by 40 countries, but not the US. Objective: To evaluate agalsidase alfa in therapy of Fabry disease. Methods: An examination of relevant reports. Results/Conclusions: Clinical trials data, along with experience of the treatment collected through participation of treating physicians in a world-wide Fabry disease registry, have demonstrated that it improves pain and stabilizes renal function, as well as cardiomyopathy, in some patients. More data are needed to evaluate the role of treatment with this drug in the prevention of stroke and adverse cardiac events, and its overall effect on the lifespan and quality of life of affected individuals.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据