Importance Sickle cell disease (SCD) is a life-threatening genetic disorder affecting nearly
100 000 individuals in the United States and is associated with many acute and chronic …

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 …

Hydroxyurea has many characteristics of an ideal drug for sickle cell anemia (SCA) and
provides therapeutic benefit through multiple mechanisms of action. Over the past 25 years …

Background Sickle-cell anaemia is associated with substantial morbidity from acute
complications and organ dysfunction beginning in the first year of life. Hydroxycarbamide …

Previous studies have determined the short-term toxicity profile, laboratory changes, and
clinical efficacy associated with hydroxyurea (HU) therapy in adults with sickle cell anemia …

Abstract Background Deoxygenated sickle hemoglobin (HbS) polymerization drives the
pathophysiology of sickle cell disease. Therefore, direct inhibition of HbS polymerization has …

Purpose: Infantile glycogen storage disease type II (GSD-II) is a fatal genetic muscle
disorder caused by deficiency of acid α-glucosidase (GAA). The purpose of this study was to …

Background For children with sickle cell anaemia and high transcranial doppler (TCD) flow
velocities, regular blood transfusions can effectively prevent primary stroke, but must be …

Pelger–Huët anomaly (PHA; OMIM* 169400) is an autosomal dominant disorder
characterized by abnormal nuclear shape and chromatin organization in blood …

Red blood cell transfusions have reduced morbidity and mortality for patients with sickle cell
disease. Transfusions can lead to erythrocyte alloimmunization, however, with serious …