Publications by authors named "Francesca Ciotti"

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Long-Term Effects of Atidarsagene Autotemcel for Metachromatic Leukodystrophy.
Francesca Fumagalli, Valeria Calbi, Vera Gallo, Alberto Andrea Zambon, Salvatore Recupero, Francesca Ciotti

N Engl J Med· April 2025

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Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Francesca Fumagalli, Valeria Calbi, Maria Grazia Natali Sora, Maria Sessa, Cristina Baldoli, Francesca Ciotti

Lancet· January 2022

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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome.
Bernhard Gentner, Francesca Tucci, Stefania Galimberti, Francesca Fumagalli, Maurizio De Pellegrin, Francesca Ciotti

N Engl J Med· November 2021

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Metachromatic leukodystrophy: A single-center longitudinal study of 45 patients.
Francesca Fumagalli, Alberto A Zambon, Paola M V Rancoita, Cristina Baldoli, Sabrina Canale, Francesca Ciotti

J Inherit Metab Dis· September 2021

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Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy.
Valeria Calbi, Francesca Fumagalli, Giulia Consiglieri, Rachele Penati, Serena Acquati, Francesca Ciotti

Bone Marrow Transplant· July 2018

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Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.
Giulio Cossu, Stefano C Previtali, Sara Napolitano, Maria Pia Cicalese, Francesco Saverio Tedesco, Francesca Ciotti

EMBO Mol Med· December 2016

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Intra-arterial transplantation of HLA-matched donor mesoangioblasts in Duchenne muscular dystrophy.
Giulio Cossu, Stefano C Previtali, Sara Napolitano, Maria Pia Cicalese, Francesco Saverio Tedesco, Francesca Ciotti

EMBO Mol Med· December 2015

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Health related quality of life in Middle Eastern children with beta-thalassemia.
Giovanni Caocci, Fabio Efficace, Francesca Ciotti, Maria Grazia Roncarolo, Adriana Vacca

BMC Blood Disord· June 2012

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Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation.
Giovanni Caocci, Fabio Efficace, Francesca Ciotti, Maria Grazia Roncarolo, Adriana Vacca

Biol Blood Marrow Transplant· June 2011

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