Clinical Trials and Research

Gene therapies for sickle cell disease are already changing lives in high-income countries, but in places like Uganda—where the burden of disease is among the…

Sickle cell anemia is a severe inherited hemoglobinopathy with a large global burden, particularly in sub-Saharan Africa.1 Hydroxyurea, which is standard therapy…

AbstractObjectivesSickle Cell Disease (SCD), characterized by persistently low hemoglobin (Hb) and hematocrit levels, is among the leading causes of under-five…

Study OverviewBrief SummaryThis study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical…

AbstractSickle cell disease (SCD) is caused by a point mutation in the β-globin gene that promotes hemoglobin polymerization, leading to chronic hemolytic anemia,…

AbstractIntroduction: Sickle cell disease (SCD) is a genetic condition with serious complications, frequently necessitating emergency medical attention. This study…

Fertility also improved, with 2 successful pregnancies after treatment: StudyNearly every person with sickle cell disease (SCD) who underwent a new regimen for stem…