cerca CERCA
Domenica 13 Giugno 2021
Aggiornato: 00:27
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European Hematology Association - Mutations in PIGA Cause a New Form of Juvenile Hemochromatosis

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- The iron overload disorder hereditary hemochromatosis is the most common genetic disorder in the Caucasian population. If untreated, iron overload causes progressive organ damage in ageing patients, including heart failure, diabetes, liver cirrhosis and cancer. In addition to the frequent late-onset subtype of hemochromatosis, rare forms have been described with severe iron accumulation in children. Our work identifies a novel subtype of juven...

European Hematology Association - Above and BEYOND: Luspatercept is Efficacious and Well-Tolerated in Patients with Non-Transfusion-Dependent β-Thalassemia

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- The non-transfusion-dependent (NTD) form of β-thalassemia, an inherited blood disorder with impaired production of hemoglobin, is characterized by chronic anemia of mild-moderate severity and iron overload. Despite their transfusion-independence, patients with NTD β-thalassemia can still develop serious morbidities in various organ systems and have reduced quality of life. There are currently no approved therapies for the management of anemia ...

European Hematology Association - Restoration of T Helper Cell Imbalance in Immune Thrombocytopenia by β2-Adrenergic Receptor Agonists

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- Immune thrombocytopenia (ITP) is a disease manifesting as a low platelet count in which dysregulation of T helper (Th) cells play a central role. Th1 and Th17 subsets are associated with the production of autoantibodies against platelets and restoration of the immune imbalance of Th cells results in clinical improvement. Recent studies have demonstrated the presence of β2-adrenergic receptor (β2-AR) as the primary adrenergic receptor on immune...

European Hematology Association - Clinical Trial Efficacy of CAR-T Therapy in Patients with Diffuse Large B-cell Lymphoma Is Mirrored in the Real World

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- DESCAR-T is the French national registry for patients treated with commercial chimeric antigen receptor (CAR)-T cells across all hematological malignancies aimed to collect real-world data including safety and effectiveness up to 15 years after CAR-T cell infusion. Several complementary registries are also linked to the DESCAR-T database such as an immune-monitoring database, a blood and tumor biobank, and an imagery platform. In this study, w...

European Hematology Association - Genetic Modification of Autologous Hematopoietic Stem Cells: A Potential Treatment for Mucopolysaccharidosis Type I Hurler

THE HAGUE, Netherlands, June 11, 2021 /PRNewswire/ -- Mucopolysaccharidosis type I Hurler (MPS-IH) is caused by a deficiency in alpha-L-iduronidase (IDUA) and is typically managed by allogeneic hematopoietic stem cell (HSC) transplantation, which has shown high rates of success when patients are treated at a young age. However, skeletal abnormalities remain and progressive neurocognitive deterioration over time severely affect a patient's quality of life. In this first-in-human phase I/II clinica...

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