ea0077oc5.1 | Bone and Calcium | SFEBES2021
Matsumoto Tae
, Miyake Noriko
, Zhao Dongwei
, Narisawa Sonoko
, Millan Jose
, Miyake Koichi
Hypophosphatasia (HPP) is an inherited skeletal disease characterized by defective bone mineralization due to a deficiency in tissue-nonspecific alkaline phosphatase (TNALP). Patients with the severe infantile form of HPP have a poor prognosis that often results in high mortality by one year. Asfotase alfa is an approved therapy for HPP, while requires chronic injections to maintain efficacy. To develop a one-time gene therapy for HPP, we examined the safety and efficacy of AA...