A multi-country time and motion study to describe the experience and burden associated with the treatment of Fabry disease with enzyme replacement therapy with agalsidase alfa and agalsidase beta
The multi-region findings provide a more complete picture of the burden associated with ERT administration for FD treatment on patients, caregivers, and HCPs. Results may support further cost-effectiveness modelling for novel treatment approaches and inform treatment decisions and patient management.
Authors
- Yin‐Hsiu Chien (ORCID: https://orcid.org/0000-0001-8802-5728)
- Nur Arslan (ORCID: https://orcid.org/0000-0003-3151-3741)
- Dau-Ming Niu (ORCID: https://orcid.org/0000-0003-3962-1739)
- Sema Kalkan Uçar (ORCID: https://orcid.org/0000-0001-9574-7841)
- Simon Shohet (ORCID: https://orcid.org/0009-0003-0901-5233)
- Laura Baldock
- Ian Keyzor (ORCID: https://orcid.org/0000-0001-5297-0816)
- Hiroyuki Yamakawa (ORCID: https://orcid.org/0000-0003-3100-5077)
- Ana María Martins (ORCID: https://orcid.org/0000-0003-2239-0882)
- Joseph Giuliano
- Leyla Tümer
Institutions
- Amicus Therapeutics (United Kingdom) (GB)
- Dokuz Eylül University (TR)
- Keio University (JP)
- Taipei Veterans General Hospital (TW)
- Ege University (TR)
- National Taiwan University Hospital (TW)
- Amicus Therapeutics (United States) (US)
- Yokohama Municipal Citizen's Hospital (JP)
- Universidade Federal de São Paulo (BR)
- Gazi University (TR)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-08-11
- DOI
- https://doi.org/10.1186/s13023-025-03707-2
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Amicus Therapeutics
- Keio University