First-in-human single-dose study of nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal glucosylceramidase: Safety, tolerability, pharmacokinetics, an...

by Paquet Luzy, Cecile, Doppler, Emilie, Polasek, Thomas M., Giorgino, Ruben
Published in Molecular genetics and metabolism

A phase 2 clinical study in Niemann-Pick disease type C and GM2 gangliosidosis patients (RAINBOW)

by Giugliani, Roberto, Valle, Daniel A.D., Horovitz, Dafne, Martins, Ana Maria, Crapard, Laetitia, Doppler, Emilie, Hurst, Noemie, de Frutos, Laura López, Luzy, Cecile Paquet
Published in Molecular genetics and metabolism

Prospective longitudinal study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 gangliosidosis (PRONTO study)

by Giugliani, Roberto, Heron, Benedicte, Jarnes, Jeanine R., Patterson, Marc C., Scarpa, Maurizio, Doppler, Emilie, Luzy, Cecile Paquet
Published in Molecular genetics and metabolism

Effect of Hirtisation treatment on surface quality and mechanical properties of AlSi10Mg samples produced by laser powder bed fusion

by Beevers, Emilie, Neumayer, Dominic, Bonvoisin, Benoit, Brandão, Ana, Hansal, Selma, Doppler, Michael, Rohr, Thomas, Van Hooreweder, Brecht
Published in Materials today communications