Zobrazeno 1 - 10
of 21
pro vyhledávání: '"Michela, Gabaldo"'
Autor:
Anneliene Hechtelt Jonker, Liliana Batista, Michela Gabaldo, Virginie Hivert, Diego Ardigo, on behalf of the IRDiRC ODDG TF and IRDiRC TSC
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 18, Iss 1, Pp 1-7 (2023)
Abstract Drug development is a complex, resource intensive and long process in any disease area, and developing medicines to treat rare diseases presents even more challenges due to the small patient populations, often limited disease knowledge, hete
Externí odkaz:
https://doaj.org/article/ef87001399d64f61adb413120e8fe703
Autor:
Luigi Naldini, Maria Pia Cicalese, Maria Ester Bernardo, Bernhard Gentner, Michela Gabaldo, Giuliana Ferrari, Alessandro Aiuti
Publikováno v:
HemaSphere, Vol 6, Iss 2, p e671 (2022)
Externí odkaz:
https://doaj.org/article/f0a3af6429944f7aaabff2c58a1b2e5f
Autor:
Heide Stirnadel-Farrant, Mahesh Kudari, Nadia Garman, Jessica Imrie, Bikramjit Chopra, Stefania Giannelli, Michela Gabaldo, Ambra Corti, Stefano Zancan, Alessandro Aiuti, Maria Pia Cicalese, Rohit Batta, Jonathan Appleby, Mario Davinelli, Pauline Ng
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 13, Iss 1, Pp 1-10 (2018)
Abstract Background Strimvelis (autologous CD34+ cells transduced to express adenosine deaminase [ADA]) is the first ex vivo stem cell gene therapy approved by the European Medicines Agency (EMA), indicated as a single treatment for patients with ADA
Externí odkaz:
https://doaj.org/article/a8893f1115da49d982593a6f5bd7315f
Autor:
Bernhard, Gentner, Francesca, Tucci, Stefania, Galimberti, Francesca, Fumagalli, Maurizio, De Pellegrin, Paolo, Silvani, Chiara, Camesasca, Silvia, Pontesilli, Silvia, Darin, Francesca, Ciotti, Marina, Sarzana, Giulia, Consiglieri, Chiara, Filisetti, Giulia, Forni, Laura, Passerini, Daniela, Tomasoni, Daniela, Cesana, Andrea, Calabria, Giulio, Spinozzi, Maria-Pia, Cicalese, Valeria, Calbi, Maddalena, Migliavacca, Federica, Barzaghi, Francesca, Ferrua, Vera, Gallo, Simona, Miglietta, Erika, Zonari, Patali S, Cheruku, Claudia, Forni, Marcella, Facchini, Ambra, Corti, Michela, Gabaldo, Stefano, Zancan, Serena, Gasperini, Attilio, Rovelli, Jaap-Jan, Boelens, Simon A, Jones, Robert, Wynn, Cristina, Baldoli, Eugenio, Montini, Silvia, Gregori, Fabio, Ciceri, Maria G, Valsecchi, Giancarlo, la Marca, Rossella, Parini, Luigi, Naldini, Alessandro, Aiuti, Maria-Ester, Bernardo, Ilaria, Visagalli
Publikováno v:
New England Journal of Medicine. 385:1929-1940
Background Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.
Autor:
Maddalena Migliavacca, Maria Ester Bernardo, Michela Gabaldo, Elena Fratini, Maria Grazia Natali Sora, Francesco Morena, Jesus Segovia, Giada Farinelli, Alessandra Biffi, Gerald Downey, Clelia Di Serio, Federica Cugnata, Luigi Naldini, Cristina Baldoli, Sabata Martino, Francesca Tucci, Mirko Essing, Francesca Ferrua, Federica Barzaghi, Eugenio Montini, Stefano Scarparo, Andrea Calabria, Maria Pia Cicalese, Mara Sangalli, Paolo Silvani, Maddalena Fraschini, Alberto A. Zambon, Marina Sarzana, Laetitia C. Schwab, Alessandra G. Clerici, Sara Locatelli, Alessandro Aiuti, Salvatore Recupero, Stefano Zancan, Francesca Ciotti, Paola M.V. Rancoita, Fabiola De Mattia, Gigliola Antonioli, Maria Sessa, Massimo Filippi, Fabio Ciceri, Vera Gallo
Publikováno v:
Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics.
Autor:
Luigi Naldini, Maria Pia Cicalese, Maria Ester Bernardo, Bernhard Gentner, Michela Gabaldo, Giuliana Ferrari, Alessandro Aiuti
Publikováno v:
HemaSphere. 6(2)
In 2016, the European Hematology Association (EHA) published the EHA Roadmap for European Hematology Research1 aiming to highlight achievements in the diagnostics and treatment of blood disorders, and to better inform European policy makers and other
Autor:
Michela Gabaldo
Publikováno v:
Nature Italy.
The pandemic has shown how the path from the lab to the clinic can be shortened. These lessons must be applied to innovations such as stem cells and gene therapy. The pandemic has shown how the path from the lab to the clinic can be shortened. These
Autor:
Michela Gabaldo
Publikováno v:
Nature Italy.
La pandemia ha mostrato come abbreviare il passaggio dal laboratorio all'applicazione clinica. Lezioni da applicare a innovazioni come cellule staminali e terapia genica. La pandemia ha mostrato come abbreviare il passaggio dal laboratorio all'applic
Autor:
Michela Gabaldo, Diego Ardigò, Simon Day, Ken Sakushima, Annemieke Aartsma-Rus, Virginie Hivert, Daniel O’Connor, Liliana Batista, Anneliene H. Jonker
Publikováno v:
Nature Reviews Drug Discovery, 19(8), 495-496. NATURE RESEARCH
The International Rare Diseases Research Consortium (IRDiRC) has created a Guidebook to facilitate drug development for rare diseases by organizing available tools into a standardized framework. The International Rare Diseases Research Consortium (IR
Autor:
Vera Gallo, Maria Ester Bernardo, Clelia De Serio, Paula M.V. Rancoita, Gerald Downey, Stefano Zancan, Serena Acquati, Francesca Tucci, John Sharpe, Francesca Fumagalli, Cristina Baldoli, Laetitia C. Schwab, Sabata Martino, Valeria Calbi, Luigi Naldini, Alessandro Aiuti, Fabiola De Mattia, Maria Grazia Natali Sora, Maria Sessa, Alessandra Biffi, Massimo Filippi, Fabio Ciceri, Alberto A. Zambon, Michela Gabaldo, Eugenio Montini
Publikováno v:
Molecular Genetics and Metabolism. 129:S59