Publications
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Research in Sickle Cell Disease: From Bedside to Bench to Bedside
Sickle cell disease (SCD) is an exemplar of bidirectional translational research, starting with a remarkable astute observation of the abnormally shaped red blood cells that motivated decades of bench research…
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Innovative Treatments for Rare Anemias
Rare anemias (RA) are mostly hereditary disorders with low prevalence and a broad spectrum of clinical severity, affecting different stages of erythropoiesis or red blood cell components. RA often remains…
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Survival and Causes of Death in 2,033 Patients with Non-Transfusion-Dependent β-Thalassemia
Non-transfusion-dependent β-thalassemia (NTDT) is a broad term encompassing patients who do not require lifelong transfusion therapy for survival. Our understanding of the disease process in NTDT has evolved significantly over…
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Survival and Causes of Death in 2,033 Patients with Non-Transfusion-Dependent β-Thalassemia
Non-transfusion-dependent β-thalassemia (NTDT) is a broad term encompassing patients who do not require lifelong transfusion therapy for survival. Our understanding of the disease process in NTDT has evolved significantly over…
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Setting up and Sustaining Blood and Marrow Transplant Services for Children in Middle-Income Economies: An Experience-Driven Position Paper on Behalf of the EBMT PDWP
Severe blood disorders and cancer are the leading cause of death and disability from noncommunicable diseases in the global pediatric population and a major financial burden. The most frequent of…
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Hydroxyurea Use Among Children With Sickle Cell Disease at King Abdulaziz University Hospital in Jeddah City
Background Sickle cell disease (SCD) is an autosomal recessive hemoglobinopathy that is very common and causes a great burden in Saudi Arabia and worldwide. This study aims to determine the…
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Assessment of Subclinical Renal Glomerular and Tubular Dysfunction in Children with Beta Thalassemia Major
Background: A good survival rate among patients with beta thalassemia major (beta-TM) has led to the appearance of an unrecognized renal disease. Therefore, we aimed to assess the role of…
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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with severe and potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin…
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Hemoglobin disorders in Europe: A Systematic Effort of Identifying and Addressing Unmet Needs and Challenges by the Thalassaemia International Federation
Hemoglobin disorders (thalassemia and sickle cell disease) are a group of hereditary anemias that today occur across the world. The recent population movement has led to a steady increase of…
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The Use of Luspatercept for Thalassemia in Adults
Luspatercept is an activin receptor ligand trap that has been shown to enhance late-stage erythropoiesis in animal models of β-thalassemia. A multicenter, international, phase 2 dose-finding study was initiated in…
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