Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria
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DOI: 10.1371/journal.pone.0173269
Abstract: Phenylketonuria (PKU) is a genetic metabolic disease in which the decrease or loss of phenylalanine hydroxylase (PAH) activity results in elevated, neurotoxic levels of phenylalanine (Phe). Due to many obstacles, PAH enzyme replacement therapy is… read more here.
Keywords: treatment; pal; phenylalanine ammonia; ravpal peg ... See more keywords