
Autosomal dominant polycystic kidney disease (ADPKD) is associated with forming large fluid-filled renal cysts that typically cause pain from early adulthood, kidney failure by the sixth decade, and numerous extrarenal symptoms, including hypertension and hematuria. Although the progression of ADPKD is slow, it has proven difficult to identify effective treatments, with drugs assessed as either lacking efficacy or not well tolerated by patients due to challenging side effect profiles. Hence, a challenge has been to identify sufficiently broad-acting drugs to counteract the signalling defects associated with ADPKD while maintaining the high safety profile required for drugs with chronic use.
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