Asfotase Alfa Treatment in a 2-year-old Girl with Childhood Hypophosphatasia

• Main Authors: Gönül Çatlı (Author), Berna Eroğlu Filibeli (Author), Handan Çelik (Author), Özlem El (Author), Bumin Dündar (Author)
• Format: Book
• Published: Galenos Yayinevi, 2022-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Childhood hypophosphatasia (HPP) presents with bowing of the limbs, poor mobility, chronic pain, short stature, fractures, and motor impairment. Enzyme replacement therapy (ERT) provides improved pulmonary and physical function in life-threatening perinatal and infantile forms of HPP. However, treat...