Six-year clinical outcomes of enzyme replacement therapy for perinatal lethal and infantile hypophosphatasia in Korea: Two case reports.
Insung KimEu-Seon NohMin-Sun KimJa-Hyun JangTae Yeon JeonHae Won ChoiSung Yoon ChoPublished in: Medicine (2023)
Among the 2 patients, the patient who started ERT early had a much better prognosis despite a more severe initial clinical presentation. Our results suggest that early diagnosis and prompt treatment play an important role in improving long-term prognosis and avoiding morbidity and premature mortality in patients with perinatal and infantile HPP.