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Hematopoietic stem cell gene therapy for mucopolysaccharidosis type I-Hurler syndrome (OTL-203): Interim skeletal, neurological and systemic outcomes from a phase I/II study Bernardo, Maria Ester M.; Tucci, Francesca; Consiglieri, Giulia; Oficialdegui, Maria Luz Uria; Fumagalli, Francesca; De Pellegrin, Maurizio; Cossutta, Matilde; Filisetti, Chiara; Manitto, Maria Pia; Butera, Carla; Dane, Giulia; Camesasca, Chiara; Parini, Rossella; Riera, Mireia Del Toro; de Heredia, Cristina Diaz; Aiuti, Alessandro Share Save
Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognition, language, and speech and slows brain demyelination and atrophy in early-onset metachromatic leukodystrophy Fumagalli, Francesca; Calbi, Valeria; Zambon, Alberto Andrea; Gallo, Vera; Baldoli, Cristina; Recupero, Salvatore; Tucci, Francesca; Consiglieri, Giulia; Ciotti, Francesca; Fraschini, Maddalena; Locatelli, Sara; Facchini, Marcella; Clerici, Alessandra; Moro, Sean; Christopherso, Kent; Gollop, Nicholas; Yarzi, Muska; Nutkins, Philippa; Shenker, Andrew; Brooks, Jean; Campbell, Laura; Bernardo, Maria Ester; Aiuti, Alessandro Share Save
Gene therapy in advanced metachromatic leukodystrophy: tempering expectations Schoenmakers, Daphne H.; Beerepoot, Shanice; Adang, Laura A.; Asbreuk, Marije A. B. C.; Bergner, Caroline G.; Bley, Annette E.; Boelens, Jaap-Jan; Calbi, Valeria; Darling, Alejandra; Eklund, Erik; Garcia Cazorla, Angeles; Gronborg, Sabine W.; Groeschel, Samuel; van Hasselt, Peter M.; Hollak, Carla E. M.; Horgan, Claire; Jones, Simon; de Koning, Tom; Laugwitz, Lucia; Lindemans, Caroline; Martin, Pascal; Mochel, Fanny; Oberg, Andreas; Ram, Dipak; Sevin, Caroline; Schoels, Ludger; Zerem, Ayelet; Wolf, Nicole, I; Fumagalli, Francesca Share Save
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy Adang, Laura Ann; Groeschel, Samuel; Grzyb, Chloe; D'Aiello, Russell; Gavazzi, Francesco; Sherbini, Omar; Bronner, Nowa; Patel, Akshilkumar; Vincent, Ariel; Sevagamoorthy, Anjana; Mutua, Sylvia; Muirhead, Kayla; Schmidt, Johanna; Pizzino, Amy; Yu, Emily; Jin, Danielle; Eichler, Florian; Fraser, Jamie L.; Emrick, Lisa; Van Haren, Keith; Boulanger, Jean-Martin; Ruzhnikov, Maura; Sylvain, Michel; Nguyen, Cam-Tu Emilie; Potic, Ana; Keller, Stephanie; Fatemi, Ali; Uebergang, Eloise; Poe, Michele; Yazdani, Pouneh Amir; Bernat, John; Lindstrom, Kristen; Bonkowsky, Joshua L.; Bernard, Genevieve; Stutterd, Chloe A.; Orchard, Paul; Gupta, Ashish O.; Ljungberg, Merete; Groenborg, Sabine; Zambon, Alberto; Locatelli, Sara; Fumagalli, Francesca; Elguen, Saskia; Kehrer, Christiane; Krageloh-Mann, Ingeborg; Shults, Justine; Vanderver, Adeline; Escolar, Maria L. Share Save
Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States Adang, Laura A.; Bonkowsky, Joshua L.; Boelens, Jaap Jan; Mallack, Eric; Ahrens-Nicklas, Rebecca; Bernat, John A.; Bley, Annette; Burton, Barbara; Darling, Alejandra; Eichler, Florian; Eklund, Erik; Emrick, Lisa; Escolar, Maria; Fatemi, Ali; Fraser, Jamie L.; Gaviglio, Amy; Keller, Stephanie; Patterson, Marc C.; Orchard, Paul; Orthmann-Murphy, Jennifer; Santoro, Jonathan D.; Schoels, Ludger; Sevin, Caroline; Srivastava, Isha N.; Rajan, Deepa; Rubin, Jennifer P.; Van Haren, Keith; Wasserstein, Melissa; Zerem, Ayelet; Fumagalli, Francesca; Laugwitz, Lucia; Vanderver, Adeline Share Save
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome 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 Share Save
Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries Schoenmakers, Daphne H.; Mochel, Fanny; Adang, Laura A.; Boelens, Jaap-Jan; Calbi, Valeria; Eklund, Erik A.; Gronborg, Sabine W.; Fumagalli, Francesca; Groeschel, Samuel; Lindemans, Caroline; Sevin, Caroline; Schoels, Ludger; Ram, Dipak; Zerem, Ayelet; Graessner, Holm; Wolf, Nicole I. Share Save
Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD) 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 Share Save
Validation of GMFC-MLD scale as a measure of gross motor function in metachromatic leukodystrophy Mutua, Sylvia; Sevagamoorthy, Anjana; Gavazzi, Francesco; Thakur, Nivedita; Woidill, Sarah; Yu, Emily; Fumagalli, Francesca; Groeschel, Samuel; Bernard, Genevieve; Stutterd, Chloe; Kehrer, Christiane; Emrick, Lisa; Shults, Justine; Vanderver, Adeline; Adang, Laura A. Share Save
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 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 Share Save
Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report Faccioli, Silvia; Sassi, Silvia; Pandarese, Daniela; Borghi, Corrado; Montemaggiori, Valentina; Sarzana, Marina; Scarparo, Stefano; Butera, Carla; Calbi, Valeria; Aiuti, Alessandro; Fumagalli, Francesca Share Save
Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi) Schoenmakers, Daphne H.; Beerepoot, Shanice; van den Berg, Sibren; Adang, Laura; Bley, Annette; Boelens, Jaap-Jan; Fumagalli, Francesca; Goettsch, Wim G.; Gronborg, Sabine; Groeschel, Samuel; van Hasselt, Peter M.; Hollak, Carla E. M.; Lindemans, Caroline; Mochel, Fanny; Mol, Peter G. M.; Sevin, Caroline; Zerem, Ayelet; Schols, Ludger; Wolf, Nicole, I Share Save
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 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 Share Save
Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System Pontesilli, Silvia; Baldoli, Cristina; Della Rosa, Pasquale Anthony; Cattoni, Alessandro; Bernardo, Maria Ester; Meregalli, Pamela; Gasperini, Serena; Motta, Serena; Fumagalli, Francesca; Tucci, Francesca; Baciga, Federica; Consiglieri, Giulia; Canonico, Francesco; De Lorenzo, Paola; Chiapparini, Luisa; Gentner, Bernhard; Aiuti, Alessandro; Biondi, Andrea; Rovelli, Attilio; Parini, Rossella Share Save
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