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
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. METHODS: We are conducting an ongoing study involving eight children with MPSIH. At enrollment, the children lacked a suitable allogeneic donor and had a Developmental Quotient or Intelligence Quotient score above 70 (i...
November 18, 2021: New England Journal of Medicine