Mechanistic modelling of enzyme-restoration effects of new recombinant liver-targeted proteins in acute intermittent porphyria.
Diego Vera-YuncaKarol M CórdobaZinnia P Parra-GuillenDaniel JericóAntonio FontanellasIñaki F TrocónizPublished in: British journal of pharmacology (2022)
The model developed had excellent performance over a range of doses and formulation type. This mechanistic model warrants use beyond ApoAI-conjugates and represents a useful tool towards more efficient drug treatments of other enzymopenias as well as for acute intermittent porphyria.