1-(13)C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)

PURPOSE: To develop a safe and noninvasive in vivo assay of hepatic propionate oxidative capacity. METHODS: A modified 1-(13)C-propionate breath test was administered to 57 methylmalonic acidemia (MMA) subjects, including 19 transplant recipients, and 16 healthy volunteers. Isotopomer enrichment ((1...

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Detalles Bibliográficos
Autores principales: Manoli, Irini, Pass, Alexandra R., Harrington, Elizabeth A., Sloan, Jennifer L., Gagné, Jack, McCoy, Samantha, Bell, Sarah L., Hattenbach, Jacob D., Leitner, Brooks P., Duckworth, Courtney J., Fletcher, Laura A., Cassimatis, Thomas M., Galarreta, Carolina I., Thurm, Audrey, Snow, Joseph, Van Ryzin, Carol, Ferry, Susan, Mew, Nicholas Ah, Shchelochkov, Oleg A., Chen, Kong Y., Venditti, Charles P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8354855/
https://www.ncbi.nlm.nih.gov/pubmed/33820958
http://dx.doi.org/10.1038/s41436-021-01143-8

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