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

Koeberl D, Schulze A, Sondheimer N, Lipshutz GS, Geberhiwot T, Li L, Saini R, Luo J, Sikirica V, Jin L, Liang M, Leuchars M, Grunewald S. Interim analyses of a first-in-human phase 1/2 mRNA trial for propionic acidaemia. Nature. 2024 Apr 3. doi: 10.1038/s41586-024-07266-7. Epub ahead of print. PMID: 38570682.


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