Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA

Abstract

This study demonstrates a novel application of a robust statistical method to evaluate outcomes in long-term intervention studies where placebo comparisons are not feasible, providing wide application for rare diseases. This comparison with real-world natural history data suggests that maralixibat improves event-free survival in patients with ALGS.