The burden of Alagille syndrome: uncovering the potential of emerging therapeutics – a comprehensive systematic literature review

Aim: Alagille syndrome (ALGS) is a rare, cholestatic multiorgan disease associated with bile duct paucity, leading to cholestasis. Clinical symptoms of cholestasis include debilitating pruritus, xanthomas, fatsoluble vitamin deficiencies, growth failure, renal disease and impaired health-related q...

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Main Authors: Philip Bufler, Robin Howard, Lucia Quadrado, Guy Lacey, Jolan Terner-Rosenthal, Andrea Goldstein, Pamela Vig, Deirdre Kelly
Format: Article
Language:English
Published: Becaris Publishing Limited 2025-01-01
Series:Journal of Comparative Effectiveness Research
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