A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Patients' and caregivers' assessments

Autor: Giugliani, Roberto, Harmatz, Paul, Héron, Bénédicte, Patterson, Marc, Schneider, Susanne, Bourchany, Aurélie, Hahn, Andreas, Valle, Daniel A.D., Barone, Rita Maria Elisa, Chabrol, Brigitte, Ardissone, Anna, Batzios, Spyros, Scarpa, Maurizio, Carp, Nicoleta, Crapard, Laetitia, de Frutos, Laura López, Quintela, Renata Medinaceli, Thiers, Aurelien, Luzy, Cecile Paquet
Zdroj: In Molecular Genetics and Metabolism February 2024 141(2)
Databáze: ScienceDirect
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