M
Maria Domenica Cappellini
Researcher at University of Milan
Publications - 570
Citations - 21441
Maria Domenica Cappellini is an academic researcher from University of Milan. The author has contributed to research in topics: Thalassemia & Deferasirox. The author has an hindex of 71, co-authored 518 publications receiving 18668 citations. Previous affiliations of Maria Domenica Cappellini include Centra & Vita-Salute San Raffaele University.
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Journal ArticleDOI
Clinical and histological evaluation of liver disease in patients with transfusion-dependent beta-thalassemia: A multicenter study
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Seven novel point mutations in the uroporphyrinogen decarboxylase (UROD) gene in patients with familial porphyria cutanea tarda (f-PCT).
Maria Domenica Cappellini,F Martinez di Montemuros,Dario Tavazzi,Silvia Fargion,Antonio Pizzuti,A. Comino,T. Cainelli,Gemino Fiorelli +7 more
TL;DR: Seven novel molecular defects in the uroporphyrinogen decarboxylase gene responsible for familial porphyria cutanea tarda in Italian subjects with reduced erythrocyte URO‐D activity are described and suggest a molecular heterogeneity at the UROD locus in Italian PCT patients.
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Morbidity-free survival and hemoglobin level in non-transfusion-dependent β-thalassemia: a 10-year cohort study.
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The Believe Trial: Results of a Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Luspatercept in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions
Maria Domenica Cappellini,Vip Viprakasit,Ali T. Taher,Pencho Georgiev,Pencho Georgiev,Kevin H.M. Kuo,Thomas D. Coates,Thomas D. Coates,Ersi Voskaridou,Hong Keng Liew,Idit Pazgal-Kobrowski,Gian Luca Forni,Silverio Perrotta,Abderrahim Khelif,Ashutosh Lal,Antonis Kattamis,Efthymia Vlachaki,Raffaella Origa,Yesim Aydinok,Mohamed Bejaoui,P. Joy Ho,Lee Ping Chew,Ping Chong Bee,Soo Min Lim,Meng-Yao Lu,Adisak Tantiworawit,Penka Ganeva,Liana Gercheva,Farrukh Shah,Ellis J. Neufeld,Abderrahmane Laadem,Jeevan K. Shetty,Jun Zou,Dimana Miteva,Tatiana Zinger,Peter G. Linde,Matthew L. Sherman,Olivier Hermine,John B. Porter,Antonio Piga +39 more
TL;DR: A phase 3, randomized, double-blind, placebo-controlled study to determine the efficacy and safety of luspatercept in adult β-thalassemia patients requiring regular RBC transfusions in adults, finding that the drug binds to select TGFβ superfamily ligands to reduce aberrant Smad2/3 signaling and enhance late-stage erythropoiesis.
Journal ArticleDOI
Luspatercept for the treatment of anaemia in non-transfusion-dependent β-thalassaemia (BEYOND): a phase 2, randomised, double-blind, multicentre, placebo-controlled trial.
Ali T. Taher,Maria Domenica Cappellini,Antonis Kattamis,Ersi Voskaridou,Silverio Perrotta,Antonio Piga,Aldo Filosa,John B. Porter,Thomas J. Coates,Gian Luca Forni,Alexis A. Thompson,Immacolata Tartaglione,Khaled M. Musallam,Jay Backstrom,Oriana Esposito,Ana Carolina Giuseppi,Wen Ling Kuo,Dimana O. Miteva,Jennifer Lord-Bessen,Aylin Yucel,Tatiana Zinger,Jeevan K. Shetty,Vip Viprakasit +22 more
TL;DR: Luspatercept is a potential treatment for adult patients with non-transfusion-dependent β-thalassaemia, for whom effective approved treatment options are scarce as mentioned in this paper .