Affiliation:
1. Department of Drugs and Medicines, School of Pharmaceutical Sciences, Sao Paulo State University (UNESP), Araraquara, Brazil
Abstract
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.
Funder
Conselho Nacional de Desenvolvimento Científico e Tecnológico
Fundação de Amparo à Pesquisa do Estado de São Paulo
Publisher
Bentham Science Publishers Ltd.
Subject
Pharmacology,Molecular Medicine,Drug Discovery,Biochemistry,Organic Chemistry
Cited by
8 articles.
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