Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates.

Gut · 2025 · PMC11874285 · PMID 39366725

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a clear trendno p-value reported

One week after the last drug dose, serum precursor levels showed preattack values, but there was a clear trend towards PBG accumulation in the CSF of untreated animals ( online supplemental figure 1J ).

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