SURE

Holida, Myrl, Linhart, Aleš, Longo, Nicola, Wallace, Eric, Tøndel, Camilla, Hughes, Derralynn, Warnock, David G., Pisani, Antonio, Eyskens, François, Deegan, Patrick, Feldt-Rasmussen, Ulla, Goker-Alpan, Ozlem, Mehta, Ankit, Piotti, Giovanni, Fichera, Vito, Wang, Meng, Chertkoff, Raul, Waldek, Stephen, Wilcox, William R. and Bernat, John A. (2026) 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. Orphanet Journal of Rare Diseases, 21 (1): 185. p. 185. ISSN 1750-1172

Holida, Myrl, Linhart, Aleš, Pisani, Antonio, Longo, Nicola, Eyskens, François, Goker‐Alpan, Ozlem, Wallace, Eric, Deegan, Patrick, Tøndel, Camilla, Feldt‐Rasmussen, Ulla, Hughes, Derralynn, Sakov, Anat, Rocco, Rossana, Almon, Einat Brill, Alon, Sari, Chertkoff, Raul, Warnock, David G., Waldek, Stephen, Wilcox, William R. and Bernat, John A. (2024) A phase III, open‐label clinical trial evaluating pegunigalsidase alfa administered every 4 weeks in adults with Fabry disease previously treated with other enzyme replacement therapies. Journal of Inherited Metabolic Disease. ISSN 1573-2665

Germain, Dominique P., Brand, Eva, Burlina, Alessandro, Cecchi, Franco, Garman, Scott C., Kempf, Judy, Laney, Dawn A., Linhart, Aleš, Maródi, László, Nicholls, Kathy, Ortiz, Alberto, Pieruzzi, Federico, Shankar, Suma P., Waldek, Stephen, Wanner, Christoph and Jovanovic, Ana (2018) Phenotypic characteristics of the p.Asn215Ser (p.N215S) GLA mutation in male and female patients with Fabry disease: A multicenter Fabry Registry study. Molecular Genetics & Genomic Medicine, 6 (4). pp. 492-503. ISSN 2324-9269