First successful outcomes of pegvaliase (PALYNZIQ) in children

First successful outcomes of pegvaliase (PALYNZIQ) in children

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...

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Bibliographic Details
Main Authors: Majid Alfadhel, Rayyan Albarakati
Format: Article
Language:English
Published: BMC 2024-03-01
Series:BMC Medical Genomics
Subjects:
Phenylketonuria
Pegvaliase
PALYNZIQ®
Pediatric
Case Report
Phenylalanine hydroxylase, PAH, PKU
Online Access:https://doi.org/10.1186/s12920-024-01847-1

Internet

https://doi.org/10.1186/s12920-024-01847-1

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