Reference health
Long-term comparative effectiveness of pegvaliase versus standard of care comparators in adults with phenylketonuria
36/36 checkable references clean · checked 2026-07-22
Every reference with a DOI in the deposited reference list resolved to a known work in Crossref or DataCite at the dated check, and none carried a retraction, withdrawal, or removal notice.
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The 36 checked references that resolve
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Key European guidelines for the diagnosis and management of patients with phenylketonuria
resolves10.1038/gim.2013.157
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
resolves10.1016/j.ymgme.2011.08.017
Diagnosis, classification, and genetics of phenylketonuria and tetrahydrobiopterin (BH4) deficiencies
resolves10.1016/j.ymgme.2016.04.008
Updated, web-based nutrition management guideline for PKU: An evidence and consensus based approach
resolves10.1186/s13023-015-0294-x
Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries
resolves10.1080/87565641.2016.1243109
Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With Phenylketonuria
resolves10.1016/j.ymgme.2018.09.006
Prevalence of comorbid conditions among adult patients diagnosed with phenylketonuria
resolves10.1038/ejhg.2014.114
Linking genotypes database with locus-specific database and genotype–phenotype correlation in phenylketonuria
resolves10.1517/17425255.2013.804064
Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias
resolves10.1016/j.ymgme.2019.04.004
International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria
resolves10.1007/s10545-007-0605-z
The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6<i>R</i>‐tetrahydrobiopterin): a phase II, multicentre, open‐label, screening study
resolves10.1002/ajmg.a.32562
Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria
resolves10.1007/s10545-012-9464-3
Efficacy and safety of BH4 before the age of 4 years in patients with mild phenylketonuria
resolves10.1016/S0140-6736(07)61234-3
Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study
resolves10.1016/j.ymgme.2014.11.011
A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria
resolves10.1016/j.jpeds.2008.11.040
Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study
resolves10.1016/j.ymgme.2015.02.003
Long-term safety and efficacy of sapropterin: The PKUDOS registry experience
resolves10.1016/j.ymgme.2018.03.006
Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM)
resolves10.1016/S0140-6736(13)61841-3
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
resolves10.1186/s13023-018-0858-7
Long-term safety and efficacy of pegvaliase for the treatment of phenylketonuria in adults: combined phase 2 outcomes through PAL-003 extension study
resolves10.1016/j.ymgme.2018.03.003
Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial
resolves10.1016/j.jpeds.2013.12.015
Neurocognitive Evidence for Revision of Treatment Targets and Guidelines for Phenylketonuria
resolves10.1016/j.ymgme.2017.01.001
Adherence to clinic recommendations among patients with phenylketonuria in the United States
resolves10.1111/j.1365-2788.2012.01568.x
Costs and outcomes over 36 years of patients with phenylketonuria who do and do not remain on a phenylalanine‐restricted diet
resolves10.1016/S0140-6736(02)09334-0
How practical are recommendations for dietary control in phenylketonuria?
resolves10.1016/j.ymgme.2018.06.010
Induction, titration, and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria
resolves10.1016/j.ebiom.2018.10.038
Association of immune response with efficacy and safety outcomes in adults with phenylketonuria administered pegvaliase in phase 3 clinical trials
resolves10.1080/00273171.2011.568786
An Introduction to Propensity Score Methods for Reducing the Effects of Confounding in Observational Studies
resolves10.1016/j.ymgme.2013.09.015
Evaluation of quality of life in PKU before and after introducing tetrahydrobiopterin (BH4); a prospective multi-center cohort study
resolves10.1186/1477-7525-11-218
Longitudinal quality of life analysis in a phenylketonuria cohort provided sapropterin dihydrochloride
resolves10.1038/bcj.2016.84
Blinatumomab vs historical standard therapy of adult relapsed/refractory acute lymphoblastic leukemia
The 3 references without a DOI — listed, not checked
no DOI — not checked10.1016/j.ymgme.2019.07.018_bb0055
no DOI — not checked10.1016/j.ymgme.2019.07.018_bb0110
no DOI — not checked10.1016/j.ymgme.2019.07.018_bb0170
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