A phase 2 clinical study in Niemann-Pick disease type C and GM2 gangliosidosis patients (RAINBOW) von Giugliani, Roberto, Valle, Daniel A.D., Horovitz, Dafne, Martins, Ana Maria, Crapard, Laetitia, Doppler, Emilie, Hurst, Noemie, de Frutos, Laura López, Luzy, Cecile Paquet

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

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A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Baseline clinical data von 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, Crapard, Laetitia, de Frutos, Laura López, Quintela, Renata Medinaceli, Meyer, Elisa, Thiers, Aurelien, Luzy, Cecile Paquet

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A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidosis (PRONTO): Evaluation of different assessments von 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, Crapard, Laetitia, de Frutos, Laura López, Quintela, Renata Medinaceli, Thiers, Aurelien, Luzy, Cecile Paquet

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