The Evolving Landscape of Drug Therapies for Sickle Cell Disease.
The Evolving Landscape of Drug Therapies for Sickle Cell Disease.
复制标题
镰状细胞病药物治疗的演变。
DOI：
10.1016/j.hoc.2022.06.008
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发表时间：
2022
期刊：
影响因子：
--
通讯作者：
Ataga,KennethI
中科院分区：
文献类型：
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作者：
Rai,Parul;Desai,PayalC;Ataga,KennethI
尽管镰状细胞病（SCD）可以通过异基因造血干细胞移植以及可能的基因治疗和基因编辑来治愈，但这些治疗对大多数患者来说仍然不可用。随着对疾病病理生理学的了解的增加，在开发药物疗法方面正在取得进展。羟基脲、L-谷氨酰胺、crizanlizumab和voxelotor目前已获美国食品和药物管理局批准，
Although sickle cell disease (SCD) can be cured using allogeneic hematopoietic stem cell transplantation and possibly gene therapy and gene editing, these treatments remain unavailable to most patients. As understanding of disease pathophysiology increases, progress is being made in developing drug therapies. Hydroxyurea, L-glutamine, crizanlizumab, and voxelotor are currently approved by the US Food and