Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency

por Thurberg, Beth L., Wasserstein, Melissa P., Jones, Simon A., Schiano, Thomas D., Cox, Gerald F., Puga, Ana Cristina
Publicado 2016

Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months

por Wasserstein, Melissa P., Diaz, George A., Lachmann, Robin H., Jouvin, Marie-Hélène, Nandy, Indrani, Ji, Allena J., Puga, Ana Cristina
Publicado 2018

Skeletal improvement in patients with Gaucher disease type 1: a phase 2 trial of oral eliglustat

por Kamath, Ravi S., Lukina, Elena, Watman, Nora, Dragosky, Marta, Pastores, Gregory M., Arreguin, Elsa Avila, Rosenbaum, Hanna, Zimran, Ari, Aguzzi, Rasha, Puga, Ana Cristina, Norfleet, Andrea M., Peterschmitt, M. Judith, Rosenthal, Daniel I.
Publicado 2014

Quantitative Systems Pharmacology Modeling of Acid Sphingomyelinase Deficiency and the Enzyme Replacement Therapy Olipudase Alfa Is an Innovative Tool for Linking Pathophysiology a...

por Kaddi, Chanchala D., Niesner, Bradley, Baek, Rena, Jasper, Paul, Pappas, John, Tolsma, John, Li, Jing, van Rijn, Zachary, Tao, Mengdi, Ortemann‐Renon, Catherine, Easton, Rachael, Tan, Sharon, Puga, Ana Cristina, Schuchman, Edward H., Barrett, Jeffrey S., Azer, Karim
Publicado 2018