Role of carglumic acid in the long-term management of propionic and methylmalonic acidurias
Sufin Yap, Serena Gasperini, Shirou Matsumoto, François Feillet · 2024
Read Role of carglumic acid in the long-term management of propionic and methylmalonic acidurias by Sufin Yap, Serena Gasperini, Shirou Matsumoto, François Feillet (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-11654332.txt.
Description
Yap and co-authors weigh carglumic acid for long-term care of two rare acid buildup disorders. Propionic and methylmalonic acidurias stem from faults in one metabolic path. Both can trigger high ammonia through a side fault in the urea cycle.
Carglumic acid copies a natural activator of the urea cycle's first step. It is approved to treat high ammonia in both disorders and is well tolerated. Its short-term use in acute episodes is well proven. This expert review searched PubMed for chronic use proof. It also weighs diet, ammonia scavengers, transplant, and gene therapy in long-term care.
Catalog record
| CREATOR | Sufin Yap, Serena Gasperini, Shirou Matsumoto, François Feillet |
|---|---|
| YEAR | 2024 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 46 KB |
| VERSION | 1.0 |
| LICENSE | CC BY 4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |