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pro vyhledávání: '"Jaap-Jan Boelens"'
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.