Catherine Skari
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Mar- 2021 -15 MarchScientific Literature
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…
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Feb- 2021 -20 FebruaryScientific Literature
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…
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3 FebruaryScientific Literature
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…
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Jan- 2021 -21 JanuaryScientific Literature
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…
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12 JanuaryScientific Literature
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.…
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8 JanuaryScientific Literature
The Prevention of Thalassemia Revisited: A Historical and Ethical Perspective by the Thalassemia International Federation
Hemoglobinopathies are the most common monogenic disorders in humans; among them, thalassemia constitutes a serious medical and public health problem…
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5 JanuaryScientific Literature
Nutrition in Thalassemia: A Systematic Review of Deficiency, Relations to Morbidity, and Supplementation Recommendations
Reports of nutritional deficiencies in patients with thalassemia (Thal) are common. Despite its importance, however, nutritionally focused research in Thal…
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Sep- 2020 -30 SeptemberScientific Literature
Fair Pricing of Innovative Medicines: An EHA Position Paper
High prices keep innovative medicines out of reach for many patients across Europe, resulting in growing inequalities in accessibility and…
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11 SeptemberScientific Literature
Gene Therapy of the Hemoglobinopathies
Sickle cell disease and the ß-thalassemias are caused by mutations of the ß-globin gene and represent the most frequent single…
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Aug- 2020 -27 AugustScientific Literature
Challenging the Erythropoiesis Paradigm in β-Thalassemia
β-thalassemia is a genetic disorder due to defective β-globin synthesis that results in severe anemia. A hallmark of the disease…
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