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F

Francesca Fumagalli

IRCCS San Raffaele Scientific Institute
H指数:17
被引数:2,839

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Allogeneic haematopoietic stem cell transplantation in late-onset metachromatic leukodystrophy

异基因造血干细胞移植在迟发性异染性脑白质营养不良中的应用

Brain
IF11.7
2026-09-210
PRE
Daphne H Schoenmakers; Mareen R Datema; Shanice Beerepoot; Marije A B C Asbreuk; Caroline G Bergner; Carolin Awißus; Annette Bley; Helen Sigel; Janna Bredow; Valeria Calbi; Francesca Fumagalli; Emilia Inverso; Erik A Eklund; Sabine W Grønborg; Wietske H M Grol; Cecilie Videbæk; Christiane Kehrer; Marjo S van der Knaap; Nick Schnermann; Hendrik Rosewich; Peter Lang; Peter M van Hasselt; Caroline A Lindemans; Moniek de Witte; Mirjam Langeveld; Ludger Schöls; Jaap Jan Boelens; Päivi Vieira; Johanna Uusimaa; Fanny Mochel; Caroline Sevin; Carla E M Hollak; Samuel Groeschel; Nicole I Wolf
Preliminary Data of the First Year of Newborn Screening for Metachromatic Leukodystrophy (MLD) in Lombardy

意大利伦巴第地区新生儿筛查中异染性脑白质营养不良（MLD）第一年的初步数据

International Journal of Neonatal Screening
IF4
2026-07-020
OAAI
Alessandra Vasco; Andrea Meta; Clarissa Berardo; Davide Camerlengo; Francesca Fumagalli; Davide Tonduti; Iris Fiamingo; Cristina Montrasio; Diana Postorivo; Manuela Rizzetto; Sabrina Fede; MLD-NBS Study Group; Roberto Bozic; Francisco Ferron; Valeria Calbi; Gianvincenzo Zuccotti; Alessandro Aiuti; Giancarlo la Marca; Stephana Carelli; Cristina Cereda
Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program

托斯卡纳地区进行异染性脑白质营养不良的新生儿筛查：一个成功的预防医学项目的范例

International Journal of Neonatal Screening 2025, Vol. 11, Page 30
IF4
2025-09-290
OAAI
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
Non-neurological, non skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy -OTL-203- for Hurler syndrome

非神经性、非骨骼结局：用于Hurler综合征的造血干细胞及祖细胞-基因疗法-OTL-203

Molecular Therapy
IF12
2025-09-270
PRE
Francesca Tucci; M.Luz Uria Oficialdegui; Giulia Consiglieri; Matilde Cossutta; Chiara Filisetti; Francesca Fumagalli; Carla Butera; Roberto Santangelo; Michele Colombo; Maria Pia Manitto; Monica Stoppani; Elisabetta Martina; Giulia Danè; Chiara Camesasca; Giulia Risca; Maurizio De Pellegrin; Stefano Scarparo; Marina Sarzana; Cristina Puricelli; Stefania Galimberti; Silvia Darin; Paolo Silvani; Sonia Bonanomi; Serena Gasperini; Luigi Naldini; Bernhard Gentner; Rossella Parini; Mireia Del Toro; Cristina Diaz-de-Heredia; Alessandro Aiuti; Maria Ester Bernardo
Newborn Screening for Metachromatic Leukodystrophy in Tuscany: The Paradigm of a Successful Preventive Medicine Program

托斯卡纳地区异染性脑白质营养不良的新生儿筛查：一个成功预防医学项目的范例

International Journal of Neonatal Screening 2025, Vol. 11, Page 30
IF4
2025-08-210
PRE
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
ARSA Variants Associated With Cognitive Decline and Long-Term Preservation of Motor Function in Metachromatic Leukodystrophy

ARSA变体与认知能力下降及异染性脑白质营养不良中运动功能的长期维持相关

Journal of Inherited Metabolic Disease
IF3.8
2025-08-020
OA
Shanice Beerepoot; Daphne H. Schoenmakers; Francesca Fumagalli; Samuel Groeschel; Ludger Schöls; Raphael Schiffmann; Sheila Wong; Odile Boespflug-Tanguy; Caroline Sevin; Yann Nadjar; Annette Bley; Fanny Mochel; Morten A. Horn; Cristina Baldoli; Sara Locatelli; Holger Hengel; Lucia Laugwitz; Carla E. M. Hollak; Volkmar Gieselmann; Marjo S. van der Knaap; Nicole I. Wolf
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
MOLECULAR GENETICS AND METABOLISM
IF3.5
2025-02-010
PRE
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
Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognition, language, and speech and slows brain demyelination and atrophy in early-onset metachromatic leukodystrophy
MOLECULAR GENETICS AND METABOLISM
IF3.5
2025-02-010
PRE
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
Gene therapy in advanced metachromatic leukodystrophy: tempering expectations
PROTEIN & CELL
IF12.8
2024-11-280
OAAI
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
Long-term lineage commitment in haematopoietic stem cell gene therapy

造血干细胞基因治疗中的长期谱系承诺

Nature
IF48.5
2024-10-230
OAAI
Andrea Calabria; Giulio Spinozzi; Daniela Cesana; Elena Buscaroli; Fabrizio Benedicenti; Giulia Pais; Francesco Gazzo; Serena Scala; Maria Rosa Lidonnici; Samantha Scaramuzza; Alessandra Albertini; Simona Esposito; Francesca Tucci; Daniele Canarutto; Maryam Omrani; Fabiola De Mattia; Francesca Dionisio; Stefania Giannelli; Sarah Marktel; Francesca Fumagalli; Valeria Calbi; Sabina Cenciarelli; Francesca Ferrua; Bernhard Gentner; Giulio Caravagna; Fabio Ciceri; Luigi Naldini; Giuliana Ferrari; Alessandro Aiuti; Eugenio Montini
Developmental delay can precede neurologic regression in early onset metachromatic leukodystrophy
MOLECULAR GENETICS AND METABOLISM
IF3.5
2024-08-010
PRE
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.
Consensus guidelines for the monitoring and management of metachromatic leukodystrophy in the United States
CYTOTHERAPY
IF3.2
2024-07-017
PRE
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
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome
SCIENCE TRANSLATIONAL MEDICINE
IF14.6
2024-05-012
PRE
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
Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD)
MOLECULAR GENETICS AND METABOLISM
IF3.5
2024-02-010
PRE
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
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
MOLECULAR GENETICS AND METABOLISM
IF3.5
2024-02-010
PRE
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
Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)
ORPHANET JOURNAL OF RARE DISEASES
IF3.5
2022-02-1416
OAAI
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

研究方向

暂未获取到该数据
A
Alessandro Aiuti
H 指数:78论文数:475被引数:3.2万
V
Valeria Calbi
H 指数:19论文数:81被引数:2,177
L
Luigi Naldini
H 指数:120论文数:572被引数:6.2万
M
Maria Ester Bernardo
H 指数:52论文数:227被引数:1.4万
F
Fabio Ciceri
H 指数:116论文数:1,423被引数:5.7万
F
Francesca Tucci
H 指数:16论文数:77被引数:1,647
C
Cristina Baldoli
H 指数:20论文数:120被引数:4,038
M
Marina Sarzana
H 指数:7论文数:34被引数:421
V
Vera Gallo
H 指数:18论文数:83被引数:1,352
S
Samuel Groeschel
H 指数:27论文数:138被引数:2,458