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Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia

Dwight Koeberl1, Andreas Schulze2, Neal Sondheimer2

  • 1Duke University School of Medicine, Durham, NC, USA.

Nature
|April 3, 2024
PubMed
Summary

This study evaluated mRNA-3927, a novel therapy for propionic acidemia. Preliminary results show a 70% reduction in metabolic decompensation events, indicating potential therapeutic benefit.

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