These patients develop progressive renal failure with tubulointerstitial inflammation, tubular dilatation, and mild urinary concentration defects due to accumulation of mutant UMOD protein in TAL ER.7 ADTKD-UMOD is just one of many examples of human proteinopathies for which novel therapies are sorely needed. Here, UMOD is linked to autosomal dominant medullary cystic kidney disease with or without hyperuricemia.