Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy
Long-term treatment with agalsidase alfa may provide renal, cardiac, and overall survival protection in FD.
Authors
- Derralynn Hughes (ORCID: https://orcid.org/0000-0003-4531-9173)
- Uma Ramaswami (ORCID: https://orcid.org/0000-0002-4703-7447)
- Dalia Jazukeviciene
- Jörn Schenk
- Guillem Pintos‐Morell (ORCID: https://orcid.org/0000-0002-9347-2386)
- Christina Anagnostopoulou (ORCID: https://orcid.org/0000-0003-0999-6484)
- Michael L. West (ORCID: https://orcid.org/0000-0003-4897-1378)
- Jaco Botha (ORCID: https://orcid.org/0000-0001-8974-2438)
- Kathy Nicholls (ORCID: https://orcid.org/0000-0002-1297-8722)
- Christoph Kampmann (ORCID: https://orcid.org/0000-0002-2929-2970)
- Ricardo Reisin (ORCID: https://orcid.org/0000-0002-7278-4639)
- Roberto Giugliani (ORCID: https://orcid.org/0000-0001-9655-3686)
- Dau‐Ming Niu
Institutions
- Dalhousie University (CA)
- Universidade Federal do Rio Grande do Sul (BR)
- The Royal Melbourne Hospital (AU)
- Royal Free London NHS Foundation Trust (GB)
- Johannes Gutenberg University Mainz (DE)
- Taipei Veterans General Hospital (TW)
- Takeda (Switzerland) (CH)
- University Medical Center of the Johannes Gutenberg University Mainz (DE)
- Vall d'Hebron Institut de Recerca (ES)
- National Institute on Population Medical Genetics (BR)
- Vall d'Hebron Hospital Universitari (ES)
- Hospital Británico de Buenos Aires (AR)
- University College London (GB)
Publication Details
- Journal
- Molecular Genetics and Metabolism Reports
- Published
- 2025-04-11
- DOI
- https://doi.org/10.1016/j.ymgmr.2025.101215
- Citations
- 5
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 5.82
Funders
- Takeda Pharmaceuticals International