Advances in Sickle Cell Disease Treatments

doi:10.2174/0929867327666200610175400
摘要

镰状细胞病(SCD)是一种由β -球蛋白基因单一突变引起的红细胞遗传性疾病。这一疾病主要在低收入国家折磨着全世界数百万患者，其特点是发病率高、死亡率高和预期寿命低。为了促进能够减少患者痛苦和提高其生活质量的治疗，迫切需要新的药物和非药物治疗策略。自1998年FDA批准HU以来，在发现新药方面几乎没有什么进展;然而，在过去的三年中，voxelotor, crizanlizumab和谷氨酰胺已经被批准为新的治疗替代品。此外，已经描述了新的有希望的化合物来治疗SCD的主要症状。在这里，我们以药物发现为重点，讨论了治疗SCD的新策略，这些策略在过去十年中已经被实施，发现了新的、安全的、有效的治疗方法。此外，非药理学方法，包括红细胞交换，基因治疗和造血干细胞移植将被提出。
关键词: 镰状细胞病，新药，胎儿血红蛋白，一氧化氮，抗镰状细胞，铁螯合，红细胞
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DOI https://dx.doi.org/10.2174/0929867327666200610175400	Print ISSN 0929-8673
Publisher Name Bentham Science Publisher	Online ISSN 1875-533X
当代药物化学

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