Characterizing pain in patients with Fabry disease: findings from a web-based cross-sectional survey in the US
Pain was largely reported to be triggered across all subgroups. Consistent pain profiles were noted in participants across sex and FD phenotypes. Female participants reported pain burden similar to that of male participants, and pain crisis experience was heterogeneous across the subgroups. Most participants reported improvement in symptoms after FD-specific treatment and a high treatment satisfaction score with agalsidase beta.
Authors
- Pronabesh DasMahapatra (ORCID: https://orcid.org/0000-0002-8242-0580)
- Connie Baldwin
- Jack Johnson (ORCID: https://orcid.org/0000-0003-0733-9587)
- Ibrahim Warsi (ORCID: https://orcid.org/0000-0003-3739-9046)
- Nicole Lyn (ORCID: https://orcid.org/0000-0001-9707-2446)
- Dawn A. Laney (ORCID: https://orcid.org/0000-0001-8344-8078)
- Joseph Kupferman
- Eric Wallace
Institutions
- Concordia University Wisconsin (US)
- Emory University (US)
- University of Alabama (US)
- Sanofi (France) (FR)
- Sanofi (United States) (US)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-06-16
- DOI
- https://doi.org/10.1186/s13023-025-03812-2
- Citations
- 1
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.16
Funders
- Sanofi