Induction, titration, and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria.
Sign Up to like & getrecommendations! Published in 2018 at "Molecular genetics and metabolism"
DOI: 10.1016/j.ymgme.2018.06.010
Abstract: BACKGROUND Phenylketonuria (PKU) is caused by a deficiency in phenylalanine hydroxylase enzyme activity that leads to phenylalanine (Phe) accumulation in the blood and brain. Elevated blood Phe levels are associated with complications in adults, including… read more here.
Keywords: pegvaliase; blood phe; maintenance; group ... See more keywords
International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria.
Sign Up to like & getrecommendations! Published in 2019 at "Molecular genetics and metabolism"
DOI: 10.1016/j.ymgme.2019.04.004
Abstract: Phenylketonuria (PKU) is an inherited metabolic disease caused by phenylalanine hydroxylase (PAH) deficiency. As the resulting high blood phenylalanine (Phe) concentration can have detrimental effects on brain development and function, international guidelines recommend lifelong control… read more here.
Keywords: responsiveness; sapropterin dihydrochloride; blood phe; phe ... See more keywords
Achieving efficacy in subjects with sustained pegvaliase-neutralizing antibody responses.
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DOI: 10.1016/j.ymgme.2021.09.006
Abstract: Pegvaliase (Palynziq®) is an enzyme substitution therapy using PEGylated recombinant Anabaena variabilis phenylalanine ammonia lyase (PAL) to reduce blood phenylalanine (Phe) levels in adults with phenylketonuria (PKU). In Phase 3 clinical studies, all subjects treated… read more here.
Keywords: pegvaliase neutralizing; nab titers; pegvaliase; blood phe ... See more keywords