Qualitative Analysis and Comparison of Externally Led Patient-Focused Drug Development Concepts for Autosomal Recessive Polycystic Kidney Disease Against SONG Initiatives.

ABSTRACT: Autosomal recessive polycystic kidney disease (ARPKD) is a rare, inherited condition associated with pathogenic variants in the PKHD1 gene, leading to fibrocystin dysfunction. ARPKD carries significant morbidity, involving progressive kidney dysfunction that often necessitates transplantation or dialysis. Liver complications commonly accompany renal manifestations and contribute to marked clinical heterogeneity and challenging disease management. Current therapies focus on alleviating complications rather than halting or reversing disease progression. Against this backdrop, patient-focused drug development (PFDD) has emerged to incorporate patients' perspectives and priorities into medical product development and care. On August 29, 2023, an externally led PFDD meeting brought toget

SUBMITTER: Soyfer B 

PROVIDER: S-EPMC12595366 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature