In vitro and in vivo effects of Ambroxol chaperone therapy in two Italian patients affected by neuronopathic Gaucher disease and epilepsy

por Ciana, Giovanni, Dardis, Andrea, Pavan, Eleonora, Da Riol, Rosalia Maria, Biasizzo, Jessica, Ferino, Dania, Zanatta, Manuela, Boni, Antonella, Antonini, Luisa, Crichiutti, Giovanni, Bembi, Bruno
Publicado 2020

Exploring the Pathophysiologic Cascade Leading to Osteoclastogenic Activation in Gaucher Disease Monocytes Generated via CRISPR/Cas9 Technology

por Ormazabal, Maximiliano Emanuel, Pavan, Eleonora, Vaena, Emilio, Ferino, Dania, Biasizzo, Jessica, Mucci, Juan Marcos, Serra, Fabrizio, Cifù, Adriana, Scarpa, Maurizio, Rozenfeld, Paula Adriana, Dardis, Andrea Elena
Publicado 2023