Matching clinical and genetic diagnoses in autosomal dominant polycystic kidney disease reveals novel phenocopies and potential candidate genes.
Ria SchönauerSebastian BaatzMelanie Nemitz-KliemchenValeska FrankFriederike PetzoldSebastian SewerinBernt PoppJohannes MünchSteffen NeuberCarsten BergmannJan HalbritterPublished in: Genetics in medicine : official journal of the American College of Medical Genetics (2020)
By illustrating vast genetic heterogeneity, this study demonstrates the value of genetic testing in a real-world PKD cohort by diagnostic verification, falsification, and disease prediction. In the era of specific treatment for fast progressive ADPKD, genetic confirmation should form the basis of personalized patient care.