Case-control study about the acceptance of Pegvaliase in Phenylketonuria
Open Access
- 1 March 2020
- journal article
- research article
- Published by Elsevier BV in Molecular Genetics and Metabolism Reports
- Vol. 22, 100557
- https://doi.org/10.1016/j.ymgmr.2019.100557
Abstract
No abstract availableOther Versions
This publication has 23 references indexed in Scilit:
- Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With PhenylketonuriaDevelopmental Neuropsychology, 2016
- Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countriesOrphanet Journal of Rare Diseases, 2015
- Long-term safety and efficacy of sapropterin: The PKUDOS registry experienceMolecular Genetics and Metabolism, 2015
- Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trialThe Lancet, 2014
- Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochlorideHealth and Quality of Life Outcomes, 2013
- Evaluation of quality of life in PKU before and after introducing tetrahydrobiopterin (BH4); a prospective multi-center cohort studyMolecular Genetics and Metabolism, 2013
- Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuriaJournal of Inherited Metabolic Disease, 2012
- PhenylketonuriaThe Lancet, 2010
- The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6R‐tetrahydrobiopterin): a phase II, multicentre, open‐label, screening studyJournal of Inherited Metabolic Disease, 2007
- Assessment of Tetrahydrobiopterin (BH4) Responsiveness in PhenylketonuriaThe Journal of Pediatrics, 2007