Publications by authors named "Naja Nyffenegger"
Article Synopsis
- * The study tested an oral ferroportin inhibitor called vamifeport in a mouse model (C282Y) that mimics the most common genetic mutation in humans with hemochromatosis, finding that it effectively lowered iron levels in the bloodstream and liver.
- * Chronic treatment with vamifeport not only maintained lower iron levels but did so without hindering the effects of phlebotomy, suggesting it could be a viable therapy for hemochromatosis patients who undergo iron removal.
β-thalassemia is an inherited anemia characterized by ineffective erythropoiesis. Blood transfusions are required for survival in transfusion-dependent β-thalassemia and are also occasionally needed in patients with non-transfusion-dependent β-thalassemia. Patients with transfusion-dependent b-thalassemia often have elevated transferrin saturation (TSAT) and non-transferrin-bound iron (NTBI) levels, which can lead to organ iron overload, oxidative stress, and vascular damage.
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- * Researchers hypothesized that reducing iron levels by blocking ferroportin, the iron transporter, could lower the sickle hemoglobin concentration and prevent complications.
- * In studies using the Townes mouse model, the ferroportin inhibitor vamifeport showed promising results, reducing hemolysis and inflammation, and improving blood flow, supporting its potential as a new treatment for SCD.
Intravenously administered iron-carbohydrate preparations are a structurally heterogenous class of nanomedicines. Iron biodistribution to target tissues is greatly affected by the physicochemical characteristics of these nanoparticles. Some regulatory agencies have recommended performing studies in animal models for biodistribution characterization and bioequivalence evaluation.
View Article and Find Full Text PDFIn β-thalassemia, ineffective erythropoiesis leads to anemia and systemic iron overload. The management of iron overload by chelation therapy is a standard of care. However, iron chelation does not improve the ineffective erythropoiesis.
View Article and Find Full Text PDFβ-Thalassemia is a genetic anemia caused by partial or complete loss of β-globin synthesis, leading to ineffective erythropoiesis and RBCs with a short life span. Currently, there is no efficacious oral medication modifying anemia for patients with β-thalassemia. The inappropriately low levels of the iron regulatory hormone hepcidin enable excessive iron absorption by ferroportin, the unique cellular iron exporter in mammals, leading to organ iron overload and associated morbidities.
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