ey0016.5-3 | New Therapies and Novel Therapeutic Strategies | ESPEYB16
T Khan
, KW Sinkevicius
, S Vong
, A Avakian
, MC Leavitt
, H Malanson
, A Marozsan
, KL Askew
Abstract: Dis Model Mech. 2018 Oct 1; 11(10): dmm035691.In brief: Generalized arterial calcification of infancy (GACI) is a severe, rare disease characterized by excessive calcification of large and medium sized arteries caused by homozygous loss-of-function mutations in ENPP1. Here, in Enpp1 deficient mice, treatment with recombinant hu...