Case Report: Carglumic acid accelerates ammonia clearance in a neonate with methylmalonic acidemia
Carglumic acid, an orphan drug derived from N-acetylglutamate, activates rate-limiting enzymes in the urea cycle, thereby promoting ammonia clearance and detoxification. Carglumic acid is a potent treatment option for hyperammonemia resulting from rare urea cycle disorders; however, clinical experie...
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| Autores principales: | , , , , , , |
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| Formato: | Artigo |
| Lenguaje: | Inglês |
| Publicado: |
Frontiers Media S.A.
2025-12-01
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| Colección: | Frontiers in Pediatrics |
| Materias: | |
| Acceso en línea: | https://www.frontiersin.org/articles/10.3389/fped.2025.1651216/full |
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