First successful outcomes of pegvaliase (PALYNZIQ) in children

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 concent...

Source: BMC Medical Genomics - March 21, 2024 Category: Genetics & Stem Cells Authors: Majid Alfadhel and Rayyan Albarakati Tags: Case Report Source Type: research