Voxelotor: A Ray of Hope for Sickle Disease

被引：14

作者：

AlDallal, Salma M. ^{[1
]}

机构：

[1] Amiri Hosp, Kuwait, Kuwait

来源：

CUREUS JOURNAL OF MEDICAL SCIENCE | 2020年 / 12卷 / 02期

关键词：

sickle cell disease; voxelotor; us fda;

D O I：

10.7759/cureus.7105

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

Sickle cell disease is one challenging blood disorder, affecting around 100,000 people in the United States alone. None of the currently approved drugs can modify the underlying pathology of the disease. Voxelotor, first of its kind, is an orally administered drug that can alter the underlying disease pathology (by increasing the affinity between Hb and oxygen) and inhibit sickling of red blood cells. Several clinical trials and case series have documented the benefits and safety of voxelotor therapy in sickle cell disease. Currently, the US FDA has approved the drug for treatment of sickle cell disease and also granted the status of orphan drug.

引用

页数：5

共 14 条

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