First successful outcomes of pegvaliase (PALYNZIQ) in children

• Κύριοι συγγραφείς: Majid Alfadhel (Συγγραφέας), Rayyan Albarakati (Συγγραφέας)
• Μορφή: Βιβλίο
• Έκδοση: BMC, 2024-03-01T00:00:00Z.
• Θέματα: article
• Διαθέσιμο Online: Connect to this object online.

Abstract Background PKU is an autosomal recessive hereditary inborn error of metabolism caused by a lack of phenylalanine hydroxylase enzyme activity. Pegvaliase (PALYNZIQ®) treatment has been approved to reduce blood Phe concentrations in adult phenylketonuria patients with uncontrolled blood Phe c...