Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia
Topics
Propionic acidemia · mRNA-3927 · Propionyl-CoA carboxylase · Propionyl-CoA carboxylase · Propionyl-CoA carboxylase · dual mRNA therapy · metabolic decompensation events · dose-optimization study · first-in-human phase 1/2 trial · intravenous mRNA dosing · dose escalation · dose-limiting toxicities · treatment-emergent adverse events · extension study · toxic metabolite accumulation · mRNA exposure pharmacokinetics
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Pepkio Research Index (PRI). Topics and Trends in Most Cited Metabolism and Genetic Disorders Papers, Class of 2026. https://pri.pepkio.com/top-papers/metabolism-and-genetic-disorders/2026. Accessed 2026-07-24. Methodology Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459

