| Literature DB >> 32520675 |
Aline Renata Pavan1, Jean Leandro Dos Santos1.
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. Copyright© Bentham Science Publishers; For any queries, please email at epub@benthamscience.net.Entities:
Keywords: Sickle cell disease; antisickling; fetal hemoglobin; iron chelation; new drugs; nitric oxide; red bloodzzm321990cell
Year: 2021 PMID: 32520675 DOI: 10.2174/0929867327666200610175400
Source DB: PubMed Journal: Curr Med Chem ISSN: 0929-8673 Impact factor: 4.530