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

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Orphanet journal of rare diseases - ISSN 1750-1172-21:1 (2026) p. 1-19
Auteur(s)
    Myrl Holida, Ales Linhart, Nicola Longo, Eric Wallace, Camilla Tondel, 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

High-resolution mass spectrometry method for targeted screening and monitoring of Fabry, Gaucher and ASMD using dried blood spots and capitainers : impact of sample matrix on measurement results

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International journal of molecular sciences - ISSN 1661-6596-26:15 (2025) p. 1-18

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

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Journal of inherited metabolic disease - ISSN 0141-8955-48:1 (2025) p. 1-17
Auteur(s)
    Myrl Holida, Ales Linhart, Antonio Pisani, Nicola Longo, François Eyskens, Ozlem Goker-Alpan, Eric Wallace, Patrick Deegan, Camilla Tondel, Ulla Feldt-Rasmussen, Derralynn Hughes, Anat Sakov, Rossana Rocco, Einat Brill Almon, Sari Alon, Raul Chertkoff, David G. Warnock, Stephen Waldek, William R. Wilcox, John A. Bernat

Unexplained splenomegaly as a diagnostic marker for a rare but severe disease with an innovative and highly effective new treatment option : a case report

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Molecular Genetics and Metabolism Reports - ISSN 2214-4269-41 (2024) p. 1-4

Left ventricular hypertrophy : do not forget Fabry disease. Diagnostic work-up and differential diagnosis

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Acta cardiologica - ISSN 0001-5385-79:6 (2024) p. 642-649
Auteur(s)