Endocrine Abstracts

Background: ENPP1 Deficiency is a rare disorder due to inactivating mutations in the ENPP1 gene. It is characterized by low levels of inorganic pyrophosphate (PPi), a critical regulator of mineralization; subsequent pathologic soft tissue calcification results in ~50% infant mortality and life-long musculoskeletal and cardiovascular morbidities. No targeted therapy exists for this disease. INZ-701 is a recombinant ENPP1-Fc investigational product.<p class="abstext...