Reference health
Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial
24/24 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.
8 without a DOI — not checked. A reference deposited without a DOI is never matched by title or guessed at; it stays outside the checked set, and this line discloses that.
The 24 checked references that resolve
resolves10.1038/gim.2013.157
Phenylalanine hydroxylase deficiency: diagnosis and management guideline
resolves10.1007/s10545-008-0978-7
Quality of life in noncompliant adults with phenylketonuria after resumption of the diet
resolves10.1037/neu0000358
Cognitive profile and mental health in adult phenylketonuria: A PKU-COBESO study.
resolves10.1007/s10545-011-9329-1
Correlation of age‐specific phenylalanine levels with intellectual outcome in patients with phenylketonuria
resolves10.1016/S0140-6736(02)09334-0
How practical are recommendations for dietary control in phenylketonuria?
resolves10.1016/j.ymgme.2017.01.001
Adherence to clinic recommendations among patients with phenylketonuria in the United States
resolves10.1016/j.ymgme.2010.05.017
Suboptimal outcomes in patients with PKU treated early with diet alone: Revisiting the evidence
resolves10.1080/87565641.2016.1243109
Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults 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
resolves10.1016/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
resolves10.1371/journal.pone.0173269
Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria
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
resolves10.1016/j.ymgme.2018.03.006
Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM)
resolves10.1177/2326409816669373
An Adaptation of the Profile of Mood States for Use in Adults With Phenylketonuria
resolves10.1016/j.jval.2015.01.008
Evaluation of Neuropsychiatric Function in Phenylketonuria: Psychometric Properties of the ADHD Rating Scale-IV and Adult ADHD Self-Report Scale Inattention Subscale in Phenylketonuria
resolves10.1016/j.ymgme.2010.10.003
Routine use of CANTAB system for detection of neuropsychological deficits in patients with PKU
resolves10.1016/j.jaci.2005.12.1303
Second symposium on the definition and management of anaphylaxis: Summary report—Second National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network symposium
resolves10.1111/j.2517-6161.1995.tb02031.x
Controlling the False Discovery Rate: A Practical and Powerful Approach to Multiple Testing
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
resolves10.1037/0894-4105.17.3.458
Timing is everything: Executive functions in children exposed to elevated levels of phenylalanine.
The 8 references without a DOI — listed, not checked
no DOI — not checkedPhenylalanine hydroxylase deficiency
no DOI — not checked10.1016/j.ymgme.2018.03.003_bb0030
no DOI — not checkedEvaluation of multiple dosing regimens in phase 2 studies of rAvPAL-PEG (BMN 165) for control of blood phenylalanine levels in adults with phenylketonuria
no DOI — not checkedEvaluation of an induction, titration, and maintenance dosing regimen in a phase 2 study of RAVPAL-PEG (BMN 165) for control of blood phenylalanine levels in adults with phenylketonuria (PKU)
no DOI — not checkedPhase 2 long-term pegvaliase treatment for adults with phenylketonuria: updated year 5 safety and efficacy data from the PAL-003 extension
no DOI — not checkedMedDRA® The Medical Dictionary for Regulatory Activities terminology is the international medical terminology developed under the auspices of the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH), 2015. MedDRA® trademark is owned by IFPMA on behalf of ICH
no DOI — not checked10.1016/j.ymgme.2018.03.003_bb0145
no DOI — not checked10.1016/j.ymgme.2018.03.003_bb0150
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