Long-term efficacy and safety of pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease: results from up to 5 years of the BRIGHT F51 phase III, open-label extension study

Abstract Background Enzyme replacement therapies (ERTs) approved for Fabry disease require infusions every 2 weeks (E2W). Pegunigalsidase alfa, a PEGylated ERT with a prolonged half-life vs. other ERTs, may allow extension of the dosing interval to every 4 weeks (E4W). BRIGHT F51 (NCT03614234) is an...

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Detalhes bibliográficos

Principais autores:	Myrl Holida, Aleš Linhart, Nicola Longo, Eric Wallace, Camilla Tøndel, Derralynn Hughes, David G. Warnock, Antonio Pisani, François Eyskens, Patrick Deegan, Ulla Feldt-Rasmussen, Ozlem Goker-Alpan, Ankit Mehta, Giovanni Piotti, Vito Fichera, Meng Wang, Raul Chertkoff, Stephen Waldek, William R. Wilcox, John A. Bernat
Formato:	Artigo
Idioma:	Inglês
Publicado em:	BMC 2026-03-01
coleção:	Orphanet Journal of Rare Diseases
Assuntos:	Lysosomal storage disorders Fabry disease Enzyme replacement therapy Pegunigalsidase alfa eGFR Open-label extension
Acesso em linha:	https://doi.org/10.1186/s13023-026-04303-8
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