Catherine Skari

DRUGS | EU Pulls Authorization of Adakveo for Sickle Cell Disease

The decision comes after the Committee on Medicinal Products for Human Use (CHMP) recommended in May 2023 to pull the approval following…

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COLD AGGLUTININ DISEASE | Enjaymo Improved Life Quality in Patients for 2.5 Years

“Enjaymo provides sustained and durable treatment benefits in chronic CAD, including a continued meaningful impact on patient quality of life…

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Economic and Clinical Burden of Managing Transfusion-dependent β-Thalassemia in the United States

Aims: To describe clinical complications, treatment use, healthcare resource utilization (HCRU), and costs among patients with transfusion-dependent β-thalassemia (TDT) in the…

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Efficacy and Safety of Early-start Deferiprone in Infants and Young Children with Transfusion-dependent Beta Thalassemia: Evidence for Iron Shuttling to Transferrin in a Randomized, Double-blind, Placebo-controlled, Clinical Trial (START)

Children with transfusion‐dependent thalassemia (TDT) require regular blood transfusions that, without iron‐chelation therapy, lead to iron‐overload toxicities. Current practice delays…

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Burden of Aging: Health Outcomes Among Adolescents and Young Adults With Sickle Cell Disease

Although ≈95% of children with sickle cell disease (SCD) will reach adulthood in high-income settings, adolescents and young adults (AYA) face…

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Αlpha-thalassemia Genotypes in Vietnam: A Report of 12,030 Pregnant Women and Their Husbands Performing Prenatal Screening for Alpha-thalassemia

BACKGROUND: Αlpha (α)-thalassemia is a global health concern, and improving screening methods is crucial for disease prevention. This study aimed…

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Low-dose Warfarin May Be a Life-saving Treatment Regimen in Sickle Cell Diseases

The sickled or just hardened red blood cells (RBCs)-induced capillary endothelial damage initiates at birth, and terminates with multiorgan failures…

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Scientists Develop Safer Method for Gene-Editing Therapy Delivery

Researchers have found a new method of delivering gene-editing medicines to persons with blood illnesses such as thalassaemia and sickle…

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NEW SURVEY | Thalassaemia Care Delivery From A Patient Perspective

The survey focuses on capturing the patient perspective on thalassaemia care delivery in various countries. This initiative by TIF aims…

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Deferiprone Reduces Iron Overload in Young Patients With Transfusion-Dependent Beta-Thalassaemia

In young patients with transfusion-dependent beta-thalassaemia (TDT), the use of early-start deferiprone appeared to be well tolerated and was associated…

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