The study authors have discovered that GPI-anchoring of HJV is crucial for its function in regulating hepcidin. The results demonstrate that PIGA mutations are responsible for a novel form of juvenile hemochromatosis.

The study authors conclude that treatment with luspatercept resulted in significant improvements of anemia, as measured by hemoglobin levels, versus placebo in adults with NTD β-thalassemia. Changes in NTDT-PRO T/W correlated with increases in hemoglobin. Luspatercept was well tolerated in this patient population.

The study authors suggest with their results that GT accomplishes extensive metabolic correction of peripheral and central compartments and promising preliminary clinical outcomes together with a favorable safety profile, highlighting the therapeutic potential of HSC-GT for the treatment of MPSIH.

The study authors conclude that Pazopanib was safe and effective to manage severe bleeding in Hereditary hemorrhagic telangiectasia, durably liberating all patients from transfusion dependence and normalizing hematologic parameters at doses much lower (median 100 mg daily) than the dose used to treat malignancies (800 mg daily).

The study authors conclude that impaired sympathetic innervation of the spleen and bone marrow plays a role in the pathogenesis of immune thrombocytopenia (ITP). Terbutaline treatment corrects the dysregulated polarization of T helper cells during T-cell differentiation, suggesting a potential novel therapeutic approach for patients with ITP.