Treatment Options in Congenital Disorders of Glycosylation

FRONTIERS IN GENETICS(2021)

引用 12|浏览10
暂无评分
摘要
Despite advances in the identification and diagnosis of congenital disorders of glycosylation (CDG), treatment options remain limited and are often constrained to symptomatic management of disease manifestations. However, recent years have seen significant advances in treatment and novel therapies aimed both at the causative defect and secondary disease manifestations have been transferred from bench to bedside. In this review, we aim to give a detailed overview of the available therapies and rising concepts to treat these ultra-rare diseases.
更多
查看译文
关键词
glycosylation, congenital disorder of glycosylation, treatment, drug repurposing, chaperone, substrate supplementation, cofactor
AI 理解论文
溯源树
样例
生成溯源树,研究论文发展脉络
Chat Paper
正在生成论文摘要