Record Status This is a bibliographic record of a published health technology assessment. No evaluation of the quality of this assessment has been made for the HTA database. Citation NIHR HSRIC. Pegvaliase for the treatment of hyperphenylalaninaemia in adults with phenylketonuria – first line. Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2016 Authors' conclusions Phenylketonuria (PKU) is an inherited disorder of protein metabolism that causes a problem with the phenylalanine hydroxylase enzyme. This enzyme is responsible for breaking down the amino acid phenylalanine, a chemical in protein from food, so that in PKU the amount of phenylalanine in the blood increases. Hyperphenylalaninaemia is when the levels of phenylalanine in the blood are too high. If untreated, hyperphenylalaninaemia can cause damage to the brain which can cause severe mental disability, seizures and tremors. In addition, it may lead to psychological, social and behavioural problems, and lead to depression, anxiety, phobias and low self-esteem.
Pegvaliase is a new drug which is injected into the skin daily to treat hyperphenylalaninaemia in adult patients with PKU. PKU is currently treated by adopting a protein restricted diet and taking dietary supplements, pegvaliase could potentially allow patients to relax these dietary restrictions. Indexing Status Subject indexing assigned by CRD MeSH Adult; Humans; Phenylalanine; Phenylketonurias Language Published English Country of organisation England English summary An English language summary is available. Address for correspondence NIHR Horizon Scanning Research&Intelligence Centre, University of Birmingham, Institute of Applied Health Research, Public Health building, Edgbaston, Birmingham B15 2TT
Tel: 0121 414 9077 Email: nihrhsc@contacts.bham.ac.uk AccessionNumber 32016000816 Date abstract record published 22/06/2016 |