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

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

Koeberl, D., Schulze, A., Sondheimer, N., Lipshutz, G. S., Geberhiwot, T., Li, L., Saini, R., Luo, J., Sikirica, V., Jin, L., Liang, M., Leuchars, M., & Grunewald, S. (2024). Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia. Nature, 628(8009), 872–877.

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