ENPP1 Deficiency

ENPP1 deficiency is a rare, serious and progressive genetic condition that affects blood vessels, soft tissues and bones. The condition is associated with increased cardiovascular mortality risk across all age groups, especially in infants. Additional conditions associated with early-onset generalized arterial calcification of infancy (GACI) include ABCC6 deficiency, severe rickets, and osteomalacia in children and adults.

Learn more about our active ENPP1 deficiency trials below. To view all BioMarin’s historical trials in this area, click here.

Note: INZ-701 is now known as BMN 401 following BioMarin’s acquisition of Inozyme Pharma.