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
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
Pegvaliase: a novel treatment option for adults with phenylketonuria
Sign Up to like & getrecommendations! Published in 2019 at "Current Medical Research and Opinion"
DOI: 10.1080/03007995.2018.1528215
Abstract: Abstract Objective: In May 2018, the US Food and Drug Administration approved pegvaliase-pqpz (Palynziq*), the first enzyme substitution therapy for the treatment of phenylketonuria (PKU). This article provides an overview of the mechanism of action,… read more here.
Keywords: treatment option; pegvaliase; treatment; pegvaliase novel ... See more keywords