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Autosomal Dominant Polycystic Kidney Disease

ADPKD is a genetic condition that causes small, fluid-filled sacs, called cysts, to develop in the kidneys. The cysts in the kidneys and other organs lead to the enlargement and loss of organ function. ADPKD affects approximately 400,000 people and is the leading inheritable cause of kidney failure in the US.

The diagnosis of ADPKD relies primarily upon imaging of the kidney. Typical findings include large kidneys and multiple cysts scattered throughout both kidneys. In some cases, genetic testing is required for a definitive diagnosis. In other cases, diagnosis can be confirmed by a kidney ultrasound. There is no cure for ADPKD, but there are treatments that may help control symptoms. Kidney function will gradually deteriorate until so much is lost that kidney failure may occur.

Researchers are evaluating the impact of bardoxolone methyl, an investigational drug, on kidney function in patients with ADPKD. FALCON is designed to test the effects of bardoxolone methyl on eGFR in patients with ADPKD through 2 years of treatment.