New Therapeutical Approaches
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Scientific Literature
Outcomes With Luspatercept in Patients With Β-Thalassemia: A Systematic Review and Metaanalysis
Abstract Background: β-thalassaemia is a genetic blood disorder marked by ineffective erythropoiesis and chronic anaemia, often requiring lifelong red blood cell (RBC) transfusions. Luspatercept, an erythroid maturation agent, has emerged as…
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Scientific Literature
Evaluating Mitapivat for the Treatment of Alpha or Beta Thalassemia
INTRODUCTION Thalassemia is a group of diverse genetic disorders with worldwide distribution that affect hemoglobin synthesis. Until recently, the therapeutic approach to thalassemia was symptomatic, relying on red blood cell…
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Scientific Literature
2026 Update on Clinical Trials in β-Thalassemia
Abstract The therapeutic landscape of β-thalassemia has evolved rapidly over the past decade, shifting from a historical reliance on transfusion support and iron chelation toward disease-modifying and potentially curative therapies.…
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Scientific Literature
Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence
Abstract Thalassemia is an inherited hemoglobin disorder characterized by chronic hemolytic anemia and substantial long-term healthcare needs. In β-thalassemia major, patients typically require regular red blood cell transfusions with iron…
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Scientific Literature
Novel Therapeutic Approaches in Thalassemias, Sickle Cell Disease and Other Red Cell Disorders
In this last decade, a deeper understanding of the pathophysiology of hereditary red cell disorders and the development of novel classes of pharmacologic agents have provided novel therapeutic approaches to…
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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
SLN124, a GalNAc Conjugated 19‐mer siRNA Targeting tmprss6, Reduces Plasma Iron and Increases Hepcidin Levels of Healthy Volunteers
SLN124, an N-acetylgalactosamine conjugated 19-mer short interfering RNA, is being developed to treat iron-loading anemias (including beta-thalassemia and myelodysplastic syndromes) and myeloproliferative neoplasms (polycythemia vera). Through hepatic targeting and silencing…
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Scientific Literature
Health-related Quality of Life in Patients with β-Thalassemia: Data from the Phase 3 BELIEVE Trial of Luspatercept
Background: Patients with transfusion-dependent (TD) β-thalassemia require long-term red blood cell transfusions (RBCTs) that lead to iron overload, impacting health-related quality of life (HRQoL). Methods: The impact of luspatercept, a…
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Scientific Literature
Management of Luspatercept Therapy in Patients with Transfusion-dependent β-Thalassaemia
Patients with transfusion-dependent β-thalassaemia require lifelong, regular red blood cell transfusions for survival; however, frequent blood transfusions are associated with an increased risk of iron overload, transfusion-transmitted disease and alloimmunization,…
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Scientific Literature
Emergent Treatments for β-Thalassemia and Orphan Drug Legislations
Highlights • β-THAL is one of the most prevalent blood disorders in some regions of the world. • However, given its prevalence, in the EU and the US it is classified as…
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