Oral ferroportin inhibitor VIT‐2763: First‐in‐human, phase 1 study in healthy volunteers

Oral ferroportin inhibitor ameliorates ineffective erythropoiesis in a model of β-thalassemia

(2019) Vania Manolova et al.   JOURNAL OF CLINICAL INVESTIGATION

Clinical Classification, Screening and Diagnosis for Thalassemia

(2018) Vip Viprakasit et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Thalassaemia

(2018) Ali T Taher et al.   LANCET

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Iron homeostasis: An anthropocentric perspective

(2017) Richard Coffey et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

New strategies to target iron metabolism for the treatment of beta thalassemia

(2016) Paraskevi Rea Oikonomidou et al.   Annals of the New York Academy of Sciences

Minihepcidin peptides as disease modifiers in mice affected by  -thalassemia and polycythemia vera

(2016) C. Casu et al.   BLOOD

Clinical and methodological factors affecting non-transferrin-bound iron values using a novel fluorescent bead assay

(2016) Maciej W. Garbowski et al.   Translational Research

Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with non-transfusion-dependent thalassemia syndromes

(2016) George J. Kontoghiorghes et al.   Drug Design Development and Therapy

Ineffective erythropoiesis and regulation of iron status in iron loading anaemias

(2015) Clara Camaschella et al.   BRITISH JOURNAL OF HAEMATOLOGY

A phase 1 study of ACE-536, a regulator of erythroid differentiation, in healthy volunteers

(2014) Kenneth M. Attie et al.   AMERICAN JOURNAL OF HEMATOLOGY

Modulation of Hepcidin as Therapy for Primary and Secondary Iron Overload Disorders

(2014) Paul J. Schmidt et al.   HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA

Reducing TMPRSS6 ameliorates hemochromatosis and β-thalassemia in mice

(2013) Shuling Guo et al.   JOURNAL OF CLINICAL INVESTIGATION

 -Thalassemia Intermedia: A Clinical Perspective

(2013) K. M. Musallam et al.   Cold Spring Harbor Perspectives in Medicine

Iron and hepcidin: a story of recycling and balance

(2013) C. Camaschella   Hematology-American Society of Hematology Education Program

Ferroportin-mediated iron transport: Expression and regulation

(2012) Diane M. Ward et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

An RNAi therapeutic targeting Tmprss6 decreases iron overload in Hfe-/- mice and ameliorates anemia and iron overload in murine  -thalassemia intermedia

(2012) P. J. Schmidt et al.   BLOOD

The definition and epidemiology of non-transfusion-dependent thalassemia

(2012) David J. Weatherall   BLOOD REVIEWS

Therapeutic Hemoglobin Levels after Gene Transfer in β-Thalassemia Mice and in Hematopoietic Cells of β-Thalassemia and Sickle Cells Disease Patients

(2012) Laura Breda et al.   PLoS One

How I treat thalassemia

(2011) E. A. Rachmilewitz et al.   BLOOD

Beta-thalassemia

(2010) Antonio Cao et al.   GENETICS IN MEDICINE

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Beta-thalassemia

(2010) Renzo Galanello et al.   Orphanet Journal of Rare Diseases

Levels of non-transferrin-bound iron as an index of iron overload in patients with thalassaemia intermedia

(2009) Ali Taher et al.   BRITISH JOURNAL OF HAEMATOLOGY

Iron overload and toxicity: the hidden risk of multiple blood transfusions

(2009) A. Shander et al.   VOX SANGUINIS

High nontransferrin bound iron levels and heart disease in thalassemia major

(2008) Antonio Piga et al.   AMERICAN JOURNAL OF HEMATOLOGY