Sickle cell disease (SCD) is a group of inherited disorders caused by mutations in HBB,
which encodes haemoglobin subunit β. The incidence is estimated to be between 300,000 …

Sickle cell anemia (SCA) was first described in the Western literature more than 100 years
ago. Elucidation of its molecular basis prompted numerous biochemical and genetic studies …

Study objective We provide an updated assessment of trends in sickle cell disease (SCD)–
related mortality, a significant source of mortality in the United States among black persons …

Importance Sickle cell disease (SCD) and cystic fibrosis (CF) are severe autosomal
recessive disorders associated with intermittent disease exacerbations that require …

Background Donors other than matched siblings and low-intensity conditioning regimens
are increasingly used in haematopoietic stem cell transplantation. We aimed to compare the …

Sickle cell disease (SCD) substantially alters renal structure and function, and causes
various renal syndromes and diseases. Such diverse renal outcomes reflect the uniquely …

Sickle cell syndromes, including sickle cell disease (SCD) and sickle cell trait, are
associated with multiple kidney abnormalities. Young patients with SCD have elevated …

For over 100 years, clinicians and scientists have been unravelling the consequences of the
A to T substitution in the β-globin gene that produces haemoglobin S, which leads to the …

Sickle cell disease is a common and life-threatening haematological disorder that affects
millions of people worldwide. Abnormal sickle-shaped erythrocytes disrupt blood flow in …

Background For decades, patients with sickle cell disease have had only a limited number of
therapies available. In 2019, voxelotor (1500 mg), an oral once-daily sickle haemoglobin …