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(Texas Christian University), Derralynn Hughes(Royal Free London NHS Foundation Trust), Martha R. Charney(Cytokinetics (United States)), Robert B. Colvin(Harvard University), Laura Barisoni(Duke University), Simeon A. Boyadjiev(University of California, Davis), Alona Paz(Protalix BioTherapeutics (Israel)), Kathy Nicholls(Melbourne Health), Özlem Göker-Alpan(Lysosomal and Rare Disorders Research and Treatment Center), Pilar Giraldo(Université Paris-Saclay), Mohamed G. Atta(Johns Hopkins University), Einat Brill‐Almon(Protalix BioTherapeutics (Israel)), Mali Szlaifer(Protalix BioTherapeutics (Israel)), Raul Chertkoff(Protalix BioTherapeutics (Israel)), Derlis Gonzalez(Instituto Privado de Hematología e Investigación Clínica), Charles J. Jennette(University of North Carolina at Chapel Hill), Sari Alon(Protalix BioTherapeutics (Israel)), Ahmad Tuffaha(University of Kansas Medical Center), Bonita Rup, Myrl Holida(University of Iowa), Gustavo Maegawa(Johns Hopkins Medicine)
Journal of Inherited Metabolic Disease
March 5, 2019
Cited by 137


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