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Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program by Sabrina Malvagia, Alessandra Bettiol, Margherita Porcaro, Massimo Mura, Silvia Funghini, Daniela Ombrone, Giulia Forni, Emanuela Scolamiero, Filippo Coppi, Roberta Damiano, Cristina Cereda, Simonetta Simonetti, Annalisa Lonetti, Marta Daniotti, Anna Caciotti, Amelia Morrone, Valeria Calbi, Francesca Fumagalli, Alessandro Aiuti, Elena Procopio, Renzo Guerrini, Giancarlo la Marca
Published 2025-04-01Get full text
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Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Netw... by Annarosa Soresina, Roberto Rondelli, Lucia Dora Notarangelo, Franco Locatelli, Alessandro Aiuti, Alessandra Biffi, Marco Rabusin, Claudio Pignata, Giuseppe Menna, Arcangelo Prete, Maura Faraci, Attilio Rovelli, Francesca Conti, Patrizia Bertolini, Chiara Azzari, Caterina Cancrini, Marco Zecca, Francesca Ferrua, Maria Pia Cicalese, Francesco Cecere, Laura Dotta, Baldassarre Martire, Silvia Giliani, Daniele Moratto, Cinzia Mazza, Alessandro Plebani, Luigi D. Notarangelo, Andrea Pession, Raffaele Badolato, Fulvio Fulvio
Published 2025-06-01Get full text
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Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemia by Maria Rosa Lidonnici, Giulia Chianella, Nicole Mende, Hugo P. Bastos, Matteo Barcella, Ivan Merelli, Mariangela Storto, Valentina Romeo, Francesca Tiboni, Samantha Scaramuzza, Claudia Rossi, Laura Raggi, Annamaria Aprile, Stefania Crippa, Deena Iskander, Irene Roberts, Anastasios Karamiditris, Julia Keith, Christophe Lechauve, Mitchell J. Weiss, Nicola K. Wilson, Berthold Göttgens, Maria Ester Bernardo, Fabio Ciceri, Alessandro Aiuti, Sarah Marktel, Elisa Laurenti, Giuliana Ferrari
Published 2025-07-01Get full text
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