Endocrine Abstracts

Hypophosphatasia (HPP) is a rare, inherited, metabolic disorder caused by deficient tissue-nonspecific alkaline phosphatase activity. A heterogeneous presentation in adults includes musculoskeletal symptoms, impaired physical function, and reduced health-related quality of life (HRQoL). Asfotase alfa is the only approved treatment for pediatric-onset HPP. We evaluated, in a real-world setting, the long-term effectiveness of asfotase alfa on physical function and HRQoL among ad...