Long‐term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey
This report further supports the long-term effectiveness and safety of agalsidase alfa in adults with Fabry disease.
Authors
- Derralynn Hughes (ORCID: https://orcid.org/0000-0003-4531-9173)
- Uma Ramaswami (ORCID: https://orcid.org/0000-0002-4703-7447)
- 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)
- Siddharth Jain (ORCID: https://orcid.org/0009-0001-1579-4275)
- Dau‐Ming Niu
Institutions
- Dalhousie University (CA)
- Universidade Federal do Rio Grande do Sul (BR)
- The Royal Melbourne Hospital (AU)
- National Yang Ming Chiao Tung University (TW)
- The University of Melbourne (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)
- Medical Group Management Association (US)
- Vall d'Hebron Institut de Recerca (ES)
- Takeda (United States) (US)
- Hospital Británico de Buenos Aires (AR)
- University College London (GB)
Publication Details
- Journal
- European Journal of Clinical Investigation
- Published
- 2025-11-06
- DOI
- https://doi.org/10.1111/eci.70142
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00