Journal article

Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial

Raphael Schiffmann, Ozlem Goker-Alpan, Myrl Holida, Pilar Giraldo, Laura Barisoni, Robert B Colvin, Charles J Jennette, Gustavo Maegawa, Simeon A Boyadjiev, Derlis Gonzalez, Kathy Nicholls, Ahmad Tuffaha, Mohamed G Atta, Bonita Rup, Martha R Charney, Alona Paz, Mali Szlaifer, Sari Alon, Einat Brill-Almon, Raul Chertkoff Show all

Journal of Inherited Metabolic Disease | WILEY | Published : 2019