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Rivelazione delle alterazioni proteiche epatiche nella bile atresica neonatale e infantile.

Al-Majdoub ZM, Howard M, Achour B, Barber J, Alizai N, Rostami-Hodjegan A, et al.

Clinical pharmacology and therapeutics·1 giugno 2026·DOI ↗

Pediatric populations differ from adults in drug elimination capacity. While current scaling methods account for enzyme and transporter maturation, they overlook comorbidities, such as biliary atresia (BA), a liver disease appearing within the first 2-8 weeks of life that can progress to cirrhosis. Such conditions may impair hepatic drug clearance, requiring dose adjustments. Physiologically based pharmacokinetic (PBPK) tools aim to address such cases and have been advocated to fill gaps in clinical data instead of less formalized and evidence-based guesswork. However, the paucity of systems data in rare disease populations has hindered the development of robust PBPK models. This study used

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