Baseline urinary ALA and PBG as criteria for starting pharmacologic prophylactic treatment in acute intermittent porphyria treated with givosiran
Hung-Chou Kuo, Long-Sun Ro, Chia-Ni Lin, Chun-Che Chu, Ming-Feng Liao, Hong-Shiu Chang · 2024
Read Baseline urinary ALA and PBG as criteria for starting pharmacologic prophylactic treatment in acute intermittent porphyria treated with givosiran by Hung-Chou Kuo, Long-Sun Ro, Chia-Ni Lin, Chun-Che Chu, Ming-Feng Liao, Hong-Shiu Chang (2024) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/pmc-11667067.txt.
Description
Hung-Chou Kuo and co-authors seek start rules for a preventive drug in a rare disease in 2024. Acute intermittent porphyria causes attacks of severe belly pain from a heme building fault. True attacks can be hard to tell from chronic pain. The drug givosiran can prevent attacks, but who should start it needs clear signs.
The team reviewed two patients from a Taiwan trial of the drug. Both had raised urine levels of two heme precursors plus clear attack signs before treatment. Their attack rates and lab values were tracked before and after the trial. Baseline lab levels may help doctors judge when preventive treatment is warranted.
Catalog record
| CREATOR | Hung-Chou Kuo, Long-Sun Ro, Chia-Ni Lin, Chun-Che Chu, Ming-Feng Liao, Hong-Shiu Chang |
|---|---|
| YEAR | 2024 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 30 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |