Long-Term Outcomes of Early Enzyme Replacement Therapy With Asfotase Alfa in Perinatal Benign Hypophosphatasia: Amelioration of Bone Deformities in a Young Child.

Hypophosphatasia (HPP) is a congenital skeletal dysplasia. Enzyme replacement therapy (ERT) improves survival and bone mineralization of patients with perinatal severe HPP. However, there are few reports of ERT in patients with perinatal benign HPP, and its long-term efficacy remains unclear.