Sickle Cell Disease
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Scientific Literature
Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease
BACKGROUND Sickle cell disease is characterized by chronic hemolytic anemia and recurrent severe vaso-occlusive crises. Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hematopoietic stem and progenitor cells that have been base-edited…
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Scientific Literature
Daily Zinc Supplementation for Infection Prevention in Children With Sickle Cell Anemia: The ZIPS-2 Randomized Clinical Trial
Abstract Importance: Despite existing prevention strategies, infections remain a major cause of morbidity and mortality in children in Africa with sickle cell anemia. Objective: To determine the safety and effectiveness of daily…
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Scientific Literature
Contemporary Global Burden of Sickle Cell Anaemia Under-5 and Under-20: A Systematic Review and Meta-Analysis
Abstract Sickle cell disease (SCD), a prevalent inherited non-communicable disease, remains a neglected public health priority, especially among children and adolescents in many low- and middle-income countries. The global burden…
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Scientific Literature
Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy
Abstract Gene therapy has emerged as a transformative treatment option for individuals with sickle cell disease (SCD), with recent regulatory approvals marking a pivotal shift in clinical care. However, the…
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News
NEW WHO GUIDELINES | Strengthening Global Guidance for Paediatric and Adolescent Sickle Cell Disease Care
This highly significant publication represents the first WHO normative guideline dedicated to the diagnosis, prevention, and clinical management of sickle-cell disease in children and adolescents aged 0–19 years. It addresses…
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News
SICKLE CELL DISEASE | Alzheimer’s Drug Demonstrates Potential Therapeutic Benefit
The study explores the potential of memantine, a medication that has been safely used for many years in the treatment of Alzheimer’s disease. Researchers investigated whether this well-known, affordable drug…
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TIF News
TOPICS IN FOCUS SERIES | Guidelines for the Management of Thalassaemia Syndromes
Each booklet focuses on a specific aspect of the updated guidelines for transfusion-dependent thalassaemia (TDT), non-transfusion-dependent β-thalassaemia (NTDT), and α-thalassaemia, allowing physicians from various specialties to access critical, evidence-based recommendations…
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News
SICKLE CELL DISEASE | Inflammation during Menstrual Cycle May Trigger Pain Crises
SCD is a genetic disorder in which red blood cells can become abnormally shaped (“sickle”), leading to blockages in blood vessels, tissue damage, and intense pain known as vaso-occlusive events…
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News
SICKLE CELL DISEASE | Limited Progress in Sickle Cell Disease Highlights Barriers to Treatment Access
According to the findings, there has been limited improvement in outcomes for SCD patients, in large part due to barriers in accessing first-line therapies such as hydroxyurea. Although several new…
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News
SICKLE CELL DISEASE | Intranasal Fentanyl Shows Promise for Rapid Pain Relief in Adults
Fentanyl is a powerful painkiller that has already been widely used in children with SCD and in adults with cancer or chronic pain. Researchers now believe its use in adults…
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