Sickle Cell Disease
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Scientific News
CASGEVY | 1st Gene-Editing Therapy for Thalassaemia and SCD Approved in UK
Casgevy is the first medicine to be licensed that uses the innovative gene-editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The therapy, developed by…
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Scientific Literature
Lung Function Decline in Children with Sickle Cell Disease Treated with Hydroxyurea
Sickle cell disease (SCD), the most common form of an inherited hematological disorder, is caused by a genetic variant that leads to the synthesis of an abnormal haemoglobin, HbS. Deoxygenated…
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Scientific Literature
Impact of Imatinib on Reducing the Painful Crisis in Patients with Sickle Cell Disease
Introduction: Sickle cell disease (SCD) is a common hemoglobinopathy worldwide that causes painful crises and hospitalization of patients. These attacks decrease survival and cause chronic end-organ damage in these patients. Hypothesis: For this reason,…
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Scientific Literature
Modelling the Public Health Impact of Voxelotor in the Management of Sickle Cell Disease in France
Sickle cell disease (SCD) is an inherited blood disorder in which sickle hemoglobin (HbS) polymerizes, leading to red blood cell sickling and chronic hemolytic anemia, vaso-occlusive crises, and end-organ damage…
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Scientific Literature
Management of the Sickle Cell Trait: An Opinion by Expert Panel Members
The number of individuals with the sickle cell trait exceeds 300 million worldwide, making sickle cell disease one of the most common monogenetic diseases globally. Because of the high frequency…
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News
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 a review of additional Phase III data. The EC’s decision…
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Scientific Literature
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 difficulties in establishing adult care and experience progression of disease…
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Scientific Literature
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 and sudden deaths even at childhood. Although RBCs suspensions and…
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News
PUBLICATION | Travel Guidance for Patients and Families
Welcome to ‘Travel Guidance for Patients & Families’, on the newest TIF’s publications! This practical handbook is intended specifically for travelers with thalassaemia, sickle cell disease, and rare anaemias. Our…
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News
CALL TO ALL PATIENTS | Your Voice Matters in Research!
Today, we invite you to be an active participant in ensuring that research that takes place is aligned with your specific requirements and challenges. Your firsthand experiences as patients offer…
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