New Therapeutical Approaches
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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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Scientific Literature
Does Hepcidin Tuning Have a Role Among Emerging Treatments for Thalassemia?
The treatments available for thalassemia are rapidly evolving, with major advances made in gene therapy and the modulation of erythropoiesis. The latter includes the therapeutic potential of hepcidin tuning. In…
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Scientific Literature
In Vivo Base Editing by a Single Intravenous Vector Injection for Treatment of Hemoglobinopathies
Individuals with β-thalassemia or Sickle Cell Disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% HbF appear to be symptom-free. Here, we used a non-integrating HDAd5/35++ vector expressing a…
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Scientific Literature
Luspatercept for the Treatment of Anaemia in Non-Transfusion-Dependent β-Thalassaemia (BEYOND): A Phase 2, Randomised, Double-blind, Multicentre, Placebo-controlled Trial
Background In patients with non-transfusion-dependent β-thalassaemia, haemoglobin concentrations lower than 10 g/dL are associated with a higher risk of morbidity, mortality, and impaired quality of life. No drugs are specifically approved for…
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Scientific Literature
Innovative Treatments for Rare Anemias
Rare anemias (RA) are mostly hereditary disorders with low prevalence and a broad spectrum of clinical severity, affecting different stages of erythropoiesis or red blood cell components. RA often remains…
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