Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial
- 14 April 2014
- journal article
- research article
- Published by Elsevier BV in The Lancet
- Vol. 384 (9937), 37-44
- https://doi.org/10.1016/s0140-6736(13)61841-3
Abstract
No abstract availableKeywords
This publication has 17 references indexed in Scilit:
- Phenylalanine hydroxylase deficiencyGenetics in Medicine, 2011
- Preclinical evaluation of multiple species of PEGylated recombinant phenylalanine ammonia lyase for the treatment of phenylketonuriaProceedings of the National Academy of Sciences of the United States of America, 2008
- Blood phenylalanine monitoring for dietary compliance among patients with phenylketonuria: comparison of methodsGenetics in Medicine, 2007
- School Performance in Early and Continuously Treated PhenylketonuriaPediatric Neurology, 2005
- Frontal lobe‐dependent functions in treated phenylketonuria: Blood phenylalanine concentrations and long‐term deficits in adolescents young adultsJournal of Inherited Metabolic Disease, 2003
- Age-related working memory impairments in children with prefrontal dysfunction associated with phenylketonuriaJournal of the International Neuropsychological Society, 2002
- National Institutes of Health Consensus Development Conference Statement: Phenylketonuria: Screening and Management, October 16–18, 2000PEDIATRICS, 2001
- Deficits in memory strategy use related to prefrontal dysfunction during early development: Evidence from children with phenylketonuria.Neuropsychology, 2001
- Inherited metabolic disorders in TurkeyJournal of Inherited Metabolic Disease, 1989
- Molecular structure and polymorphic map of the human phenylalanine hydroxylase geneBiochemistry, 1986