Fatty acid nitroalkenes - Multi-target agents for the treatment of sickle cell disease.

ABSTRACT: Sickle cell disease (SCD) is a hereditary hematological disease with high morbidity and mortality rates worldwide. Despite being monogenic, SCD patients display a plethora of disease-associated complications including anemia, oxidative stress, sterile inflammation, vaso-occlusive crisis-related pain, and vasculopathy, all of which contribute to multiorgan dysfunction and failure. Over the past decade, numerous small molecule drugs, biologics, and gene-based interventions have been evaluated; however, only four disease-modifying drug therapies are presently FDA approved. Barriers regarding effectiveness, accessibility, affordability, tolerance, and compliance of the current polypharmacy-based disease-management approaches are challenging. As such, there is an unmet pharmacological need for

SUBMITTER: Chowdhury FA 

PROVIDER: S-EPMC10632539 | biostudies-literature | 2023 Oct

REPOSITORIES: biostudies-literature