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Análisis intermedios de un primer ensayo de ARNm de fase 1/2 en humanos para la acidez propiónica

Dwight Koeberl1, Andreas Schulze2, Neal Sondheimer2

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

Nature
|April 3, 2024
PubMed
Resumen

En este estudio se evaluó el mRNA-3927, una nueva terapia para la acidemia propiónica. Los resultados preliminares muestran una reducción del 70% en los eventos de descompensación metabólica, lo que indica un beneficio terapéutico potencial.

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