Long-Term Safety and Efficacy of Pegunigalsidase Alfa Administered Every 4 Weeks in Fabry Disease: 2-Year Interim Results from BRIGHT51
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Submitter Only - Adam Bijou, MD; Presenting Author - John A. Bernat, MD, PhD; Co-Author - Myrl Holida, PA-C; Co-Author - Nicola Longo, MD PhD FACMG; Co-Author - Ozlem Goker-Alpan, MD; Co-Author - Eric Wallace, MD; Co-Author - Patrick Deegan, MD; Co-Author - Camilla Tondel, MD PhD; Co-Author - Francois Eyskens, MD, PhD; Co-Author - Ulla Feldt-Rasmussen, MD, DMSc; Co-Author - Derralynn Hughes, MA DPhil FRCP FRCPath; Co-Author - Antonio Pisani, MD; Co-Author - Ales Linhart, MD, DSc; Co-Author - Rossana Rocco, MD; Co-Author - Einat Almon, PhD; Co-Author - Sari Alon, MD; Co-Author - Raul Chertkoff, MD; Co-Author - David G. Warnock, MD; Co-Author - Stephen Waldek, MD, PhD; Co-Author - William R. Wilcox, MD, PhD, FACMG;
Meta Tag
Enzyme Replacement Therapy
Lysosomal Diseases
Metabolic Disorder
Therapy
X-Inactivation/X-Linked Disease
Co-Author Myrl Holida, PA-C
Co-Author Nicola Longo, MD PhD FACMG
Co-Author Ozlem Goker-Alpan, MD
Co-Author Eric Wallace, MD
Co-Author Patrick Deegan, MD
Co-Author Camilla Tondel, MD PhD
Co-Author Francois Eyskens, MD, PhD
Co-Author Ulla Feldt-Rasmussen, MD, DMSc
Co-Author Derralynn Hughes, MA DPhil FRCP FRCPath
Co-Author Antonio Pisani, MD
Co-Author Ales Linhart, MD, DSc
Co-Author Rossana Rocco, MD
Co-Author Einat Almon, PhD
Co-Author Sari Alon, MD
Co-Author Raul Chertkoff, MD
Co-Author David G. Warnock, MD
Co-Author Stephen Waldek, MD, PhD
Co-Author William R. Wilcox, MD, PhD, FACMG
Presenting Author John A. Bernat, MD, PhD
Submitter Only Adam Bijou, MD
Keywords
BRIGHT51 study
pegunigalsidase alfa
Fabry disease
long-term safety
long-term efficacy
enzyme replacement therapies
ADAs
plasma lyso-Gb3 concentrations
glomerular filtration rate
treatment burden

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