Search Results - "Baldoli, Cristina"

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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Neural language networks at birth by Perani, Daniela, Saccuman, Maria C, Scifo, Paola, Awander, Alfred, Spada, Danilo, Baldoli, Cristina, Poloniato, Antonella, Lohmann, Gabriele, Friederici, Angela D

“…The ability to learn language is a human trait. In adults and children, brain imaging studies have shown that auditory language activates a bilateral…”
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Functional specializations for music processing in the human newborn brain by Perani, Daniela, Saccuman, Maria Cristina, Scifo, Paola, Spada, Danilo, Andreolli, Guido, Rovelli, Rosanna, Baldoli, Cristina, Koelsch, Stefan

“…In adults, specific neural systems with right-hemispheric weighting are necessary to process pitch, melody, and harmony as well as structure and meaning…”
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A hierarchical procedure to select intrauterine and extrauterine factors for methodological validation of preterm birth risk estimation by Della Rosa, Pasquale Anthony, Miglioli, Cesare, Caglioni, Martina, Tiberio, Francesca, Mosser, Kelsey H H, Vignotto, Edoardo, Canini, Matteo, Baldoli, Cristina, Falini, Andrea, Candiani, Massimo, Cavoretto, Paolo

“…Etiopathogenesis of preterm birth (PTB) is multifactorial, with a universe of risk factors interplaying between the mother and the environment. It is of utmost…”
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The effects of the functional interplay between the Default Mode and Executive Control Resting State Networks on cognitive outcome in preterm born infants at 6 months of age by Della Rosa, Pasquale Anthony, Canini, Matteo, Marchetta, Elisa, Cirillo, Sara, Pontesilli, Silvia, Scotti, Roberta, Natali Sora, Maria Grazia, Poloniato, Antonella, Barera, Graziano, Falini, Andrea, Scifo, Paola, Baldoli, Cristina

“…•Preterm birth affects attention and executive control mechanisms.•Resting State Network brain organization changes with biological maturity.•Default Mode…”
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Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated bacillus Calmette-Guérin infection by Tucci, Francesca, Gallo, Vera, Barzaghi, Federica, Ferrua, Francesca, Migliavacca, Maddalena, Calbi, Valeria, Doglio, Matteo, Fratini, Elena Sophia, Karakas, Zeynep, Guner, Sukru, Zambelli, Matilde, Parisi, Cristina, Milani, Raffaella, Gattillo, Salvatore, Mazzi, Benedetta, Oltolini, Chiara, Barbera, Maurizio, Baldoli, Cristina, Cirillo, Daniela Maria, Asnaghi, Veronica, De Min, Cristina, Cicalese, Maria Pia, Ciceri, Fabio, Aiuti, Alessandro, Bernardo, Maria Ester

“…Emapalumab, a fully human anti-IFNγ monoclonal antibody, has been approved in the US as second-line treatment of primary hemophagocytic lymphohistiocytosis…”
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The maternal-fetal neurodevelopmental groundings of preterm birth risk by Miglioli, Cesare, Canini, Matteo, Vignotto, Edoardo, Pecco, Nicolò, Pozzoni, Mirko, Victoria-Feser, Maria-Pia, Guerrier, Stéphane, Candiani, Massimo, Falini, Andrea, Baldoli, Cristina, Cavoretto, Paolo I., Della Rosa, Pasquale A.

“…Altered neurodevelopment is a major clinical sequela of Preterm Birth (PTB) being currently unexplored in-utero. To study the link between fetal brain…”
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Case Report: Off-Label Liraglutide Use in Children With Wolfram Syndrome Type 1: Extensive Characterization of Four Patients by Frontino, Giulio, Raouf, Tara, Canarutto, Daniele, Tirelli, Eva, Di Tonno, Raffaella, Rigamonti, Andrea, Cascavilla, Maria Lucia, Baldoli, Cristina, Scotti, Roberta, Leocani, Letizia, Huang, Su-Chun, Meschi, Franco, Barera, Graziano, Broccoli, Vania, Rossi, Greta, Torchio, Silvia, Chimienti, Raniero, Bonfanti, Riccardo, Piemonti, Lorenzo

“…Wolfram syndrome type 1 is a rare recessive monogenic form of insulin-dependent diabetes mellitus with progressive neurodegeneration, poor prognosis, and no…”
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Expanding the spectrum of neonatal‐onset AIFM1‐associated disorders by Zambon, Alberto A., Ghezzi, Daniele, Baldoli, Cristina, Cutillo, Gianni, Fontana, Katia, Sofia, Valentina, Patricelli, Maria Grazia, Nasca, Alessia, Vinci, Stefano, Spiga, Ivana, Lamantea, Eleonora, Fanelli, Giovanna F., Sora, Maria Grazia Natali, Rovelli, Rosanna, Poloniato, Antonella, Carrera, Paola, Filippi, Massimo, Barera, Graziano

“…Abstract Objectives Pathogenic variants in AIFM1 have been associated with a wide spectrum of disorders, spanning from CMT4X to mitochondrial encephalopathy…”
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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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Prenatal Management of Congenital Human Cytomegalovirus Infection in Seropositive Pregnant Patients Treated with Azathioprine by Cavoretto, Paolo Ivo, Fornara, Chiara, Baldoli, Cristina, Arossa, Alessia, Furione, Milena, Candiani, Massimo, Rovere Querini, Patrizia, Barera, Graziano, Poloniato, Antonella, Gaeta, Gerarda, Spinillo, Arsenio, Lilleri, Daniele

“…Human cytomegalovirus (HCMV) is the leading infectious agent causing congenital disabilities. The risk of HCMV transmission to the fetus in pregnant women…”
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High incidence of severe cyclosporine neurotoxicity in children affected by haemoglobinopaties undergoing myeloablative haematopoietic stem cell transplantation: early diagnosis and prompt intervention ameliorates neurological outcome by Noè, Anna, Cappelli, Barbara, Biffi, Alessandra, Chiesa, Robert, Frugnoli, Ilaria, Biral, Erika, Finizio, Valentina, Baldoli, Cristina, Vezzulli, Paolo, Minicucci, Fabio, Fanelli, Giovanna, Fiori, Rossana, Ciceri, Fabio, Roncarolo, Maria Grazia, Marktel, Sarah

“…Neurotoxicity is a recognized complication of cyclosporine A (CSA) treatment. The incidence of severe CSA-related neurological complications following…”
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A case of melanotic desmoplastic ganglioglioma by Antonelli, Manila, Acerno, Stefania, Baldoli, Cristina, Terreni, Maria Rosaria, Giangaspero, Felice

“…We describe a case of desmoplastic infantile ganglioglioma (DIG) in a 9‐month‐old boy located in the temporal lobe. Grossly the tumor was brown and…”
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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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Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy by Biffi, Alessandra, Montini, Eugenio, Lorioli, Laura, Cesani, Martina, Fumagalli, Francesca, Plati, Tiziana, Baldoli, Cristina, Martino, Sabata, Calabria, Andrea, Canale, Sabrina, Benedicenti, Fabrizio, Vallanti, Giuliana, Biasco, Luca, Leo, Simone, Kabbara, Nabil, Zanetti, Gianluigi, Rizzo, William B., Mehta, Nalini A. L., Cicalese, Maria Pia, Casiraghi, Miriam, Boelens, Jaap J., Del Carro, Ubaldo, Dow, David J., Schmidt, Manfred, Assanelli, Andrea, Neduva, Victor, Di Serio, Clelia, Stupka, Elia, Gardner, Jason, von Kalle, Christof, Bordignon, Claudio, Ciceri, Fabio, Rovelli, Attilio, Roncarolo, Maria Grazia, Aiuti, Alessandro, Sessa, Maria, Naldini, Luigi

“…Metachromatic leukodystrophy (MLD) is an inherited lysosomal storage disease caused by arylsulfatase A (ARSA) deficiency. Patients with MLD exhibit progressive…”
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Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial by Sessa, Maria, MD, Lorioli, Laura, MD, Fumagalli, Francesca, MD, Acquati, Serena, PhD, Redaelli, Daniela, MSc, Baldoli, Cristina, MD, Canale, Sabrina, MSc, Lopez, Ignazio D, MD, Morena, Francesco, PhD, Calabria, Andrea, PhD, Fiori, Rossana, MD, Silvani, Paolo, MD, Rancoita, Paola M V, PhD, Gabaldo, Michela, MSc, Benedicenti, Fabrizio, MSc, Antonioli, Gigliola, RN, Assanelli, Andrea, MD, Cicalese, Maria Pia, MD, del Carro, Ubaldo, MD, Sora, Maria Grazia Natali, MD, Martino, Sabata, PhD, Quattrini, Angelo, MD, Montini, Eugenio, PhD, Di Serio, Clelia, Prof, Ciceri, Fabio, MD, Roncarolo, Maria Grazia, Prof, Aiuti, Alessandro, MD, Naldini, Luigi, Prof, Biffi, Alessandra, Dr

“…Summary Background Metachromatic leukodystrophy (a deficiency of arylsulfatase A [ARSA]) is a fatal demyelinating lysosomal disease with no approved treatment…”
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Tumefactive demyelinating lesions: a challenging first manifestation of multiple sclerosis by Azzimonti, Matteo, Margoni, Monica, Zanetta, Chiara, Genovese, Federica, Martinelli, Vittorio, Rocca, Maria A., Baldoli, Cristina, Moiola, Lucia, Filippi, Massimo

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A Novel KIDINS220 Pathogenic Variant Associated with the Syndromic Spastic Paraplegia SINO: An Expansion of the Brain Malformation Spectrum and a Literature Review by Bonati, Maria Teresa, Baldoli, Cristina, Taurino, Jacopo, Marchetti, Daniela, Larizza, Lidia, Finelli, Palma, Iascone, Maria

“…Identifying novel variants in very rare disease genes can be challenging when patients exhibit a complex phenotype that expands the one described, and we…”
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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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COVID‐19 cardiac involvement in a 38‐day old infant by Del Barba, Paolo, Canarutto, Daniele, Sala, Elisa, Frontino, Giulio, Guarneri, Maria Pia, Camesasca, Chiara, Baldoli, Cristina, Esposito, Antonio, Barera, Graziano

“…The spectrum of clinical manifestations of coronavirus disease 2019 in children is yet to be fully elucidated. We report the case of an infant who tested…”
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