Effectiveness and safety of enzyme replacement therapy in the treatment of Fabry disease: a Chinese monocentric real-world study
ERT could effectively stabilize or improve renal and cardiac function and relieve neuropathic pain in patients with Fabry disease, and no AE occurred during treatment, and the clinical effectiveness and safety were satisfactory.
Authors
- Zhiqing Zhang (ORCID: https://orcid.org/0000-0002-9306-3267)
- Y. Liu
- Ying Li (ORCID: https://orcid.org/0000-0002-3506-1255)
- Pei Li (ORCID: https://orcid.org/0000-0002-2870-7484)
- Songyun Zhang
Institutions
- Hebei Medical University (CN)
- Second Hospital of Hebei Medical University (CN)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-11-11
- DOI
- https://doi.org/10.1186/s13023-024-03441-1
- Citations
- 5
- Primary Topic
- Lysosomal Storage Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 1.63
Funders
- Health Commission of Hebei Province