Endocrine Abstracts

Hypophosphatasia (HPP), a heritable metabolic bone disease, results from low alkaline phosphatase (TNSALP) activity. Inorganic pyrophosphate (PPi), an inhibitor of mineralization, and pyridoxal 5′-phosphate (PLP), are substrates that accumulate in HPP. There is no approved therapy. ENB-0040, a bone-targeted, recombinant, human TNSALP improves skeletal mineralization in affected infants and children with HPP.Objective: Evaluate substrate levels and ...