hrp0092p1-21 | Bone, Growth Plate and Mineral Metabolism | ESPE2019
Simmons Jill H.
, Rush Eric T.
, Petryk Anna
, Zhou Shanggen
, Martos-Moreno Gabriel Á.
Hypophosphatasia (HPP) is a rare, inherited, systemic disease characterized by deficient tissue-nonspecific alkaline phosphatase activity. Common manifestations in children include impaired skeletal mineralization, short stature, and reduced physical function. Asfotase alfa is an enzyme replacement therapy approved for treatment of patients of any age with pediatric-onset HPP. The utility of dual X-ray absorptiometry (DXA) as a diagnostic tool or measure of treatment effective...