Disease pharmacokinetic-pharmacodynamic modelling in acute intermittent porphyria to support the development of mRNA-based therapies.
作者： Zinnia P. Parra‐Guillen , Antonio Fontanellas , Lei Jiang , Daniel Jericó , Paolo Martini
DOI: 10.1111/BPH.15040
关键词:
摘要: Background and Purpose Acute intermittent porphyria (AIP) results from haplo‐insufficiency of the porphobilinogen deaminase (PBGD) gene encoding the third enzyme in the haem …
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