Pharmacokinetic‐pharmacodynamic model of urinary δ‐aminolevulinic acid reduction after givosiran treatment in patients with acute hepatic porphyria

Abstract Givosiran, an RNA interference‐based therapeutic, is a recent addition to the limited treatment armamentarium for acute hepatic porphyria (AHP). As a small interfering RNA that is selectively taken up in the liver, both the mechanism and targeted delivery create a complex relationship betwe...

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Autores principales: Jongtae Lee (Autor), Megan Melch (Autor), Gabriel J. Robbie (Autor)
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Publicado: Wiley, 2023-06-01T00:00:00Z.
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Número de Clasificación: A1234.567
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