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
Schiffmann, Raphael, Goker-Alpan, Ozlem, Holida, Myrl, Giraldo, Pilar, Barisoni, Laura, Colvin, Robert B., Jennette, J. Charles, Maegawa, Gustavo, Boyadjiev, Simeon A., Gonzalez, Derlis, Nicholls, KatJournal:
Journal of Inherited Metabolic Disease
DOI:
10.1002/jimd.12080
Date:
March, 2019
File:
PDF, 1.26 MB
2019