A Pilot Study on Protective Effect of Ambrisentan on Proteinuria in Patients with Alport Syndrome

Alport syndrome (AS) is the most common inherited glomerular disease caused by COL4A3/4/5 mutations, which is a leading cause of kidney failure worldwide, yet the only standard care available is renin-angiotensin-aldosterone system inhibition.1 An unmet need exists for AS treatment. In disease models of AS and other glomerular diseases, the involvement of angiotensin II and endothelin-1, either individually or in concert, in mediating glomerular injury has been well documented, leading to the development of proteinuria and kidney damage.