Table 1.

Major clinical manifestations of Alström syndrome and corresponding pre-clinical effects of PBI-4050

Clinical features of ALMS	Corresponding pre-clinical effects of PBI-4050
Loss of organ function due to fibrosis involving
Heart	↓ heart fibrosis in suprarenal aortic banding in rats
Lung	↓ lung fibrosis in bleomycin-induced lung fibrosis in mice
Liver	↓ liver fibrosis in CCl4-induced liver fibrosis in rats
Kidneys (renal failure)	↓ kidney fibrosis in various animal models of kidney fibrosis
Type 2 diabetes mellitus
Early hyperinsulinemia	Reduces insulin resistance in db/db diabetic mice and db/db eNOS−/− diabetic mice
Severe insulin resistance	Normalizes glycaemia in diabetic mice
Late pancreatic failure	Maintains (early treatment) or restores (late treatment) insulin content in pancreatic islets

eNOS^−/− endothelial nitric oxide synthase knockout (mice)