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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BMC,
2024-03-01T00:00:00Z.
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