Search Results - "Sarzana, Marina"

P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up by Fumagalli, Francesca, Calbi, Valeria, De Mattia, Fabiola, Zambon, Alberto, Gallo, Vera, Recupero, Salvatore, Fratini, Elena Sophia, Ippolito, Alessia, Ciotti, Francesca, Fraschini, Maddalena, Sarzana, Marina, Scarparo, Stefano, Montini, Eugenio, Locatelli, Sara, Facchini, Marcella, Clerici, Alessandra, Morena, Francesco, Martino, Sabata, Yarzi, Muska, Moro, Sean, Shenker, Andrew, Brooks, Jean, Richardson, Alan, Meltzer, Leslie, Campbell, Laura, Aiuti, Alessandro

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P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I‐II CLINICAL TRIAL OF EX‐VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW‐UP by Tucci, Francesca, Consiglieri, Giulia, Filisetti, Chiara, De Pellegrin, Maurizio, Mellone, Renata, Fumagalli, Francesca, Darin, Silvia, Sarzana, Marina, Scarparo, Stefano, Ciotti, Francesca, Silvani, Paolo, Baldoli, Cristina, Pontesilli, Silvia, Parini, Rossella, Marca, Giancarlo, Ciceri, Fabio, Naldini, Luigi, Aiuti, Alessandro, Gentner, Bernhard, Ester Bernardo, Maria

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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 by Fumagalli, Francesca, Calbi, Valeria, Natali Sora, Maria Grazia, Sessa, Maria, Baldoli, Cristina, Rancoita, Paola Maria V, Ciotti, Francesca, Sarzana, Marina, Fraschini, Maddalena, Zambon, Alberto Andrea, Acquati, Serena, Redaelli, Daniela, Attanasio, Vanessa, Miglietta, Simona, De Mattia, Fabiola, Barzaghi, Federica, Ferrua, Francesca, Migliavacca, Maddalena, Tucci, Francesca, Gallo, Vera, Del Carro, Ubaldo, Canale, Sabrina, Spiga, Ivana, Lorioli, Laura, Recupero, Salvatore, Fratini, Elena Sophia, Morena, Francesco, Silvani, Paolo, Calvi, Maria Rosa, Facchini, Marcella, Locatelli, Sara, Corti, Ambra, Zancan, Stefano, Antonioli, Gigliola, Farinelli, Giada, Gabaldo, Michela, Garcia-Segovia, Jesus, Schwab, Laetitia C, Downey, Gerald F, Filippi, Massimo, Cicalese, Maria Pia, Martino, Sabata, Di Serio, Clelia, Ciceri, Fabio, Bernardo, Maria Ester, Naldini, Luigi, Biffi, Alessandra, Aiuti, Alessandro

“…Effective treatment for metachromatic leukodystrophy (MLD) remains a substantial unmet medical need. In this study we investigated the safety and efficacy of…”
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Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome by Gentner, Bernhard, Tucci, Francesca, Galimberti, Stefania, Fumagalli, Francesca, De Pellegrin, Maurizio, Silvani, Paolo, Camesasca, Chiara, Pontesilli, Silvia, Darin, Silvia, Ciotti, Francesca, Sarzana, Marina, Consiglieri, Giulia, Filisetti, Chiara, Forni, Giulia, Passerini, Laura, Tomasoni, Daniela, Cesana, Daniela, Calabria, Andrea, Spinozzi, Giulio, Cicalese, Maria-Pia, Calbi, Valeria, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Gallo, Vera, Miglietta, Simona, Zonari, Erika, Cheruku, Patali S, Forni, Claudia, Facchini, Marcella, Corti, Ambra, Gabaldo, Michela, Zancan, Stefano, Gasperini, Serena, Rovelli, Attilio, Boelens, Jaap-Jan, Jones, Simon A, Wynn, Robert, Baldoli, Cristina, Montini, Eugenio, Gregori, Silvia, Ciceri, Fabio, Valsecchi, Maria G, la Marca, Giancarlo, Parini, Rossella, Naldini, Luigi, Aiuti, Alessandro, Bernardo, Maria-Ester

“…Eight patients with Hurler syndrome who lacked suitable allogeneic donors received autologous hematopoietic stem and progenitor cells transduced ex vivo with…”
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Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD) by Gallo, Vera, Calbi, Valeria, De Mattia, Fabiola, Zambon, Alberto Andrea, Recupero, Salvatore, Fratini, Elena Sophia, Ciotti, Francesca, Fraschini, Maddalena, Sangalli, Mara, Sarzana, Marina, Clerici, Alessandra, Morena, Francesco, Martino, Sabata, Brooks, Jean, Richardson, Alan, Campbell, Laura, Aiuti, Alessandro, Fumagalli, Francesca

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Metachromatic leukodystrophy: A single‐center longitudinal study of 45 patients by Fumagalli, Francesca, Zambon, Alberto A., Rancoita, Paola M. V., Baldoli, Cristina, Canale, Sabrina, Spiga, Ivana, Medaglini, Stefania, Penati, Rachele, Facchini, Marcella, Ciotti, Francesca, Sarzana, Marina, Lorioli, Laura, Cesani, Martina, Natali Sora, Maria Grazia, Del Carro, Ubaldo, Cugnata, Federica, Antonioli, Gigliola, Recupero, Salvatore, Calbi, Valeria, Di Serio, Clelia, Aiuti, Alessandro, Biffi, Alessandra, Sessa, Maria

“…In this study, we characterize the natural course of metachromatic leukodystrophy (MLD), explore intra/inter group differences, and identify biomarkers to…”
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Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up by Fumagalli, Francesca, Calbi, Valeria, De Mattia, Fabiola, Zambon, Alberto Andrea, Gallo, Vera, Recupero, Salvatore, Fratini, Elena Sophia, Ippolito, Alessia, Ciotti, Francesca, Fraschini, Maddalena, Sarzana, Marina, Scarparo, Stefano, Montini, Eugenio, Locatelli, Sara, Facchini, Marcella, Clerici, Alessandra, Morena, Francesco, Martino, Sabata, Yarzi, Muska, Moro, Sean, Shenker, Andrew, Brooks, Jean, Richardson, Alan, Campbell, Laura, Aiuti, Alessandro

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Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome by Consiglieri, Giulia, Tucci, Francesca, De Pellegrin, Maurizio, Guerrini, Barbara, Cattoni, Alessandro, Risca, Giulia, Scarparo, Stefano, Sarzana, Marina, Pontesilli, Silvia, Mellone, Renata, Gasperini, Serena, Galimberti, Stefania, Silvani, Paolo, Filisetti, Chiara, Darin, Silvia, Forni, Giulia, Miglietta, Simona, Santi, Ludovica, Facchini, Marcella, Corti, Ambra, Fumagalli, Francesca, Cicalese, Maria Pia, Calbi, Valeria, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Gallo, Vera, Recupero, Salvatore, Canarutto, Daniele, Doglio, Matteo, Tedesco, Lucia, Volpi, Nicola, Rovelli, Attilio, la Marca, Giancarlo, Valsecchi, Maria Grazia, Zancan, Stefano, Ciceri, Fabio, Naldini, Luigi, Baldoli, Cristina, Parini, Rossella, Gentner, Bernhard, Aiuti, Alessandro, Bernardo, Maria Ester

“…Mucopolysaccharidosis type I Hurler (MPSIH) is characterized by severe and progressive skeletal dysplasia that is not fully addressed by allogeneic…”
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Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use by Calbi, Valeria, Fumagalli, Francesca, Gallo, Vera, Recupero, Salvatore, De Mattia, Fabiola, Fratini, Elena Sophia, Corti, Ambra, Tarantola, Giulia, Sarzana, Marina, Ippolito, Alessia, Scarparo, Stefano, Ciotti, Francesca, Fraschini, Maddalena, Levi, Margherita, Sangalli, Mara, Morena, Francesco, Martino, Sabata, Sevin, Caroline, Narayanan, Vinodh, Adam, Etai, Emrick, Lisa, Giugliani, Roberto, Aiuti, Alessandro

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Long-term clinical outcomes of atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up by Fumagalli, Francesca, Calbi, Valeria, De Mattia, Fabiola, Zambon, Alberto A., Gallo, Vera, Recupero, Salvatore, Baldoli, Cristina, Fratini, Elena Sophia, Ciotti, Francesca, Fraschini, Maddalena, Sarzana, Marina, Scarparo, Stefano, Calabria, Andrea, Montini, Eugenio, Locatelli, Sara, Facchini, Marcella, Clerici, Alessandra, Morena, Francesco, Martino, Sabata, Claros, Angelica, Kaviya, Arpeat, Moro, Sean, Brooks, Jean, Richardson, Alan, Aiuti, Alessandro

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The Italian Consensus Conference on the role of rehabilitation for children and adolescents with leukemia, central nervous system tumors, and bone cancer, part 2: general principles for the rehabilitation treatment of motor function impairments by Rossi, Francesca, Botti, Stefano, Morri, Mattia, Asaftei, Sebastian, Bertin, Daniele, Breggiè, Simona, Casalaz, Roberto, Cervo, Marta, Ciullini, Paola, Coppo, Monica, Cornelli, Annalisa, Esposito, Maria, Ferrarese, Miriana, Ghetti, Marina, Longo, Lucia, Naretto, Gabriella, Orsini, Nicoletta, Panzeri, Daniele, Pellegrini, Chiara, Peranzoni, Michela, Perna, Antonella, Petit, Nadine, Picone, Fabiola, Pittorru, Gianna, Raffa, Debora, Recchiuti, Veronica, Rizzato, Lucia, Sarzana, Marina, Sensi, Raffaella, Fagioli, Franca, Ricci, Federica

“…In Italy, 1400 children and 800 adolescents are diagnosed with cancer every year. About 80% of them can be cured but are at high risk of experiencing severe…”
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First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction by Tucci, Francesca, Gentner, Bernhard, Galimberti, Stefania, Fumagalli, Francesca, Consiglieri, Giulia, Filisetti, Chiara, Darin, Silvia, Sarzana, Marina, Scarparo, Stefano, Ciotti, Francesca, De Pellegrin, Maurizio, Silvani, Paolo, Baldoli, Cristina, Pontesilli, Silvia, Parini, Rossella, Gasperini, Serena, Serafini, Marta, Rovelli, Attilio, Forni, Giulia, la Marca, Giancarlo, Ciceri, Fabio, Naldini, Luigi, Aiuti, Alessandro, Bernardo, Maria Ester

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Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report by Faccioli, Silvia, Sassi, Silvia, Pandarese, Daniela, Borghi, Corrado, Montemaggiori, Valentina, Sarzana, Marina, Scarparo, Stefano, Butera, Carla, Calbi, Valeria, Aiuti, Alessandro, Fumagalli, Francesca

“…(1) Background: Atidarsagene autotemcel is a hematopoietic stem and progenitor cell gene therapy (HSPC-GT) approved to treat early-onset metachromatic…”
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Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy by Fumagalli, Francesca, Calbi, Valeria, Sessa, Maria, Zambon, Alberto, Fratini, Elena, Cicalese, Maria Pia, Gallo, Vera, Ciotti, Francesca, Sarzana, Marina, Facchini, Marcella, Locatelli, Sara, Antonioli, Gigliola, Zancan, Stefano, Segovia, Jesus, Schwab, Laetitia C., Downey, Gerald, Ciceri, Fabio, Filippi, Massimo, Sora, Maria Grazia Natali, Bernardo, Maria Ester, Naldini, Luigi, Biffi, Alessandra, Aiuti, Alessandro

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Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200 by Calbi, Valeria, Fumagalli, Francesca, De Mattia, Fabiola, Fratini, Elena, Ferrua, Francesca, Barzaghi, Federica, Cicalese, Maria Pia, Migliavacca, Maddalena, Tucci, Francesca, Gallo, Vera, Ciotti, Francesca, Fraschini, Maddalena, Sarzana, Marina, Locatelli, Sara, Sangalli, Mara, Morena, Francesco, Segovia, Jesus, Schwab, Laetitia C., Downey, Gerald, Martino, Sabata, Ciceri, Fabio, Bernardo, Maria Ester, Naldini, Luigi, Aiuti, Alessandro

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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 by Calbi, Valeria, Fumagalli, Francesca, Consiglieri, Giulia, Penati, Rachele, Acquati, Serena, Redaelli, Daniela, Attanasio, Vanessa, Marcella, Facchini, Cicalese, Maria Pia, Migliavacca, Maddalena, Barzaghi, Federica, Ferrua, Francesca, Assanelli, Andrea, Silvani, Paolo, Zoccolillo, Matteo, Chidini, Giovanna, Chiesa, Robert, Arora, Ruchi, Ciotti, Francesca, Sarzana, Marina, Antonioli, Gigliola, Baldoli, Cristina, Morena, Francesco, Martino, Sabata, Ardissino, Gian Luigi, Sora, Maria Grazia Natali, Naldini, Luigi, Ciceri, Fabio, Aiuti, Alessandro, Bernardo, Maria Ester

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Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report by Faccioli, Silvia, Sassi, Silvia, Pandarese, Daniela, Borghi, Corrado, Montemaggiori, Valentina, Sarzana, Marina, Scarparo, Stefano, Butera, Carla, Calbi, Valeria, Aiuti, Alessandro, Fumagalli, Francesca

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