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Publication Information

Public Release Type
Conference Presentation
Publication Year
2023
Affiliation
1 Critical Path Institute, Tucson, AZ; 2 Sanofi, Cambridge, MA; 3 Tufts Medical Center, Boston MA
Authors
Aggarwal V, Cui Z, DasMahapatra P, Fedeles S, Leiser R, Miller C, Morales JF, Perrone R, Quinlan L, Romero K, Vanasco W, Zaph S
Studies

Abstract

Despite the regulatory advances in ADPKD including the qualification of total kidney volume (TKV) as a prognostic enrichment biomarker and its designation by the FDA as a reasonably likely surrogate endpoint, the clinical development paradigms for PKD remain challenging. This highlights the need to quantify the time course of renal decline and disease progression patterns and their sources of variability to facilitate the development of novel therapies.