Qualitative Analysis and Comparison of Externally Led Patient-Focused Drug Development (EL-PFDD) Concepts for Autosomal Recessive Polycystic Kidney Disease (ARPKD) Against SONG Initiatives
Soyfer B, Fedeles S, Ruyle W, Hoover E, Liebau M, Hartung E, Dell K, Guay-Woodford L, Perrone R, Oberdhan D. Qualitative Analysis and Comparison of Externally Led Patient-Focused Drug Development (EL-PFDD) Concepts for Autosomal Recessive Polycystic Kidney Disease (ARPKD) Against SONG Initiatives. Kidney Medicine 2025, 101110. DOI: 10.1016/j.xkme.2025.101110.Peer-Reviewed Original ResearchAutosomal recessive polycystic kidney diseasePatient-focused drug developmentRecessive polycystic kidney diseaseQuality of lifePolycystic kidney diseasePatient perspectiveARPKD patientsKidney diseaseProgressive kidney dysfunctionEarly-onset diseaseClinical trial designDrug developmentRenal manifestationsOutcome measuresLiver complicationsHepatic complicationsStandardized outcomesNovel therapiesKidney dysfunctionPathogenic variantsPKHD1 geneDisease burdenDisease managementDisease progressionClinical heterogeneity
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