P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*

Autor: Fumagalli, Francesca, Calbi, Valeria, De Mattia, Fabiola, Zambon, Alberto, 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, Meltzer, Leslie, Campbell, Laura, Aiuti, Alessandro
Zdroj: In Genetics in Medicine Open 2024 2 Supplement 1
Databáze: ScienceDirect