Baseline urinary ALA and PBG as criteria for starting pharmacologic prophylactic treatment in acute intermittent porphyria treated with givosiran
Our data from 2 AIP patients with biochemical and clinical evidence of acute attacks suggested that patient with a shorter disease duration may respond better in terms of AAR. Further studies are necessary to understand the association between disease characteristics, treatment history, and optimal treatment response for patients with recurrent AIP in terms of both attack frequency and quality of life.
Authors
- Ming‐Feng Liao (ORCID: https://orcid.org/0000-0001-7908-4267)
- Hung‐Chou Kuo (ORCID: https://orcid.org/0000-0001-8738-6476)
- Chun‐Che Chu
- Chia‐Ni Lin (ORCID: https://orcid.org/0000-0002-2722-0164)
- Hong‐Shiu Chang
- Long‐Sun Ro
Institutions
- Chang Gung University (TW)
- Chang Gung Memorial Hospital (TW)
- Linkou Chang Gung Memorial Hospital (TW)
Publication Details
- Journal
- Molecular Genetics and Metabolism Reports
- Published
- 2024-12-01
- DOI
- https://doi.org/10.1016/j.ymgmr.2024.101169
- Citations
- 2
- Primary Topic
- Porphyrin Metabolism and Disorders
- Type
- article
- Field-Weighted Citation Impact
- 0.47
Funders
- National Science and Technology Council