Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease

202327 citationsJournal Articlehybrid Open Access

Authors

Derralynn Hughes · University College London

Derlis Gonzalez · Instituto Privado de Hematología e Investigación Clínica

Gustavo Maegawa · Morgan Stanley Children's Hospital

John Bernat · University of Iowa Hospitals and Clinics

Myrl Holida · University of Iowa Hospitals and Clinics

Pilar Giraldo · Centro de Investigación Biomédica en Red

Mohamed G. Atta

Abstract

In this first long-term assessment of pegunigalsidase alfa administration in patients with FD, we found favorable safety/efficacy. Our data suggest long-term continuous benefits of pegunigalsidase alfa treatment in adults with FD.

Topics & Keywords

Lysosomal Storage Disorders Research Carbohydrate Chemistry and Synthesis Trypanosoma species research and implications

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Genetics in Medicine

Volume 25, Issue 12, pp. 100968-100968

DOI: 10.1016/j.gim.2023.100968

Field-Weighted Citation Impact: 4.79