Sickle Cell Disease (SCD) is an inherited disorder of red blood cells that is caused by a single mutation in the β -globin gene. The disease, which afflicts millions of patients worldwide mainly in low income countries, is characterized by high morbidity, mortality and low life expectancy. The new pharmacological and non-pharmacological strategies for SCD is urgent in order to promote treatments able to reduce patient’s suffering and improve their quality of life. Since the FDA approval of HU in 1998, there have been few advances in discovering new drugs; however, in the last three years voxelotor, crizanlizumab, and glutamine have been approved as new therapeutic alternatives. In addition, new promising compounds have been described to treat the main SCD symptoms. Herein, focusing on drug discovery, we discuss new strategies to treat SCD that have been carried out in the last ten years to discover new, safe, and effective treatments. Moreover, non-pharmacological approaches, including red blood cell exchange, gene therapy and hematopoietic stem cell transplantation will be presented.

镰状细胞病（SCD）是由红血球的遗传性疾病引起的，由β-珠蛋白基因中的单个突变引起。该病主要在低收入国家困扰全球数百万患者，其特点是发病率高，死亡率高，预期寿命短。为了促进能够减轻患者痛苦并改善其生活质量的治疗方法，SCD的新药理学和非药理学策略迫在眉睫。自从1998年FDA批准HU以来，发现新药的进展很少。然而，在最近三年中，voxelotor，crizanlizumab和谷氨酰胺已被批准为新的治疗选择。另外，已经描述了新的有前途的化合物来治疗主要的SCD症状。在这里，着重于药物发现，我们将讨论最近十年来为发现SCD的新策略，以发现新的，安全的和有效的治疗方法。此外，将介绍非药理学方法，包括红细胞交换，基因治疗和造血干细胞移植。