Impact of enzyme replacement therapy and migalastat on disease progression in females with fabry disease
We conclude that treatment of females with agalsidase-beta, agalsidase-alfa, and migalastat was safe. Independent of the chosen treatment regimen, nearly all patients presented with a stable disease course over time. In our cohort, a comparison of therapy efficacies showed no relevant clinical differences between the groups.
Authors
- Albina Nowak (ORCID: https://orcid.org/0000-0002-4119-5516)
- Jessica Kaufeld (ORCID: https://orcid.org/0000-0001-7718-3464)
- Nicole Muschol (ORCID: https://orcid.org/0000-0003-4213-7294)
- Markus Cybulla (ORCID: https://orcid.org/0000-0001-7414-3096)
- Christine Kurschat (ORCID: https://orcid.org/0000-0002-3646-9471)
- Malte Lenders (ORCID: https://orcid.org/0000-0001-8409-2886)
- Anja Köhn
- Eva Brand (ORCID: https://orcid.org/0009-0004-3243-5508)
Institutions
- Universität Hamburg (DE)
- University of Cologne (DE)
- Medizinische Hochschule Hannover (DE)
- University Hospital Münster (DE)
- University Medical Center Hamburg-Eppendorf (DE)
- Medizinisches Versorgungszentrum Prof. Mathey, Prof. Schofer (DE)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2025-02-20
- DOI
- https://doi.org/10.1186/s13023-025-03600-y
- Citations
- 8
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 9.31
Funders
- Takeda Pharmaceuticals International