Background Previous global analyses, with known underdiagnosis and single cause per
death attribution systems, provide only a small insight into the suspected high population …

Somatic gene therapy will be one of the most exciting practices of genetic medicine in Africa
and is primed to offer a “new life” for persons living with sickle cell disease (SCD). Recently …

Sickle cell disease (SCD) is the most common life-threatening monogenic disorder in the
world. The disease is highly prevalent in malaria endemic areas with over 75% of patients …

Sickle cell disease (SCD) is a common condition within sub-Saharan Africa and associated
with high under-5 mortality (U5M). The American Society of Hematology instituted the …

To embrace the prospects of accurately diagnosing thousands of monogenic conditions,
predicting disease risks for complex traits or diseases, tailoring treatment to individuals' …

Sickle cell disease (SCD) is the most common clinically significant hemoglobinopathy,
characterized by painful episodes, anemia, high risk of infection, and other acute and …

Rare diseases (RD) pose serious challenges in terms of both diagnosis and treatment.
Legislation was passed in the US (1983) and in EU (2000) aimed to reverse the previous …

The enrolment of children and adolescents in health research requires that attention to be
paid to specific assent and consent requirements such as the age range for seeking assent; …

Introduction: Sickle Cell Disease (SCD) causes significant morbidity and mortality
particularly in sub-Saharan Africa (SSA) where it contributes to early childhood deaths …

Purpose Sickle cell disease (SCD) is an inherited blood disorder characterized by
unpredictable episodes of acute pain and numerous health complications. Individuals with …