Real-World Experience of Carglumic Acid for Methylmalonic and Propionic Acidurias: An Interim Analysis of the Multicentre Observational PROTECT Study
Author:
Funder
Recordati Rare Diseases
Publisher
Springer Science and Business Media LLC
Link
https://link.springer.com/content/pdf/10.1007/s40268-023-00449-z.pdf
Reference44 articles.
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2. Coude FX, Sweetman L, Nyhan WL. Inhibition by propionyl-coenzyme A of N-acetylglutamate synthetase in rat liver mitochondria: a possible explanation for hyperammonemia in propionic and methylmalonic acidemia. J Clin Invest. 1979;64(6):1544–51. https://doi.org/10.1172/JCI109614.
3. Coude FX, Ogier H, Grimber G, et al. Correlation between blood ammonia concentration and organic acid accumulation in isovaleric and propionic acidemia. Pediatrics. 1982;69(1):115–7.
4. Ah Mew N, McCarter R, Daikhin Y, Nissim I, Yudkoff M, Tuchman M. N-carbamylglutamate augments ureagenesis and reduces ammonia and glutamine in propionic acidemia. Pediatrics. 2010;126(1):e208–14. https://doi.org/10.1542/peds.2010-0008.
5. Daniotti M, la Marca G, Fiorini P, Filippi L. New developments in the treatment of hyperammonemia: emerging use of carglumic acid. Int J Gen Med. 2011;4:21–8. https://doi.org/10.2147/IJGM.S10490.
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