Starting the conversation on gene therapy for phenylketonuria: Current perspectives of patients, caregivers, and advocates

Gene-based therapies for neurodegenerative diseases

(2021) Jichao Sun et al.   NATURE NEUROSCIENCE

The Genetic Landscape and Epidemiology of Phenylketonuria

(2020) Alicia Hillert et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Delivery Approaches for Therapeutic Genome Editing and Challenges

(2020) Ilayda Ates et al.   Genes

Patient and public perspectives on cell and gene therapies: a systematic review

(2020) Olalekan Lee Aiyegbusi et al.   Nature Communications

Efficacy and safety of sapropterin dihydrochloride in patients with phenylketonuria: A meta‐analysis of randomized controlled trials

(2019) Jinghan Qu et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria

(2019) Ania C. Muntau et al.   MOLECULAR GENETICS AND METABOLISM

State-of-the-Art 2019 on Gene therapy for Phenylketonuria

(2019) Hiu Man Grisch-Chan et al.   HUMAN GENE THERAPY

Psychiatric and Cognitive Aspects of Phenylketonuria: The Limitations of Diet and Promise of New Treatments

(2019) Killian Ashe et al.   Frontiers in Psychiatry

Emotional health in early-treated adults with phenylketonuria (PKU): Relationship with cognitive abilities and blood phenylalanine

(2019) Liana Palermo et al.   JOURNAL OF CLINICAL AND EXPERIMENTAL NEUROPSYCHOLOGY

Public Acceptability of Gene Therapy and Gene Editing for Human Use: A Systematic Review

(2019) Juliette Delhove et al.   HUMAN GENE THERAPY

Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM)

(2018) Janet Thomas et al.   MOLECULAR GENETICS AND METABOLISM

Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial

(2018) Cary O. Harding et al.   MOLECULAR GENETICS AND METABOLISM

Pegvaliase: a novel treatment option for adults with phenylketonuria

(2018) Karlie C. Mahan et al.   CURRENT MEDICAL RESEARCH AND OPINION

Long-term treatment of phenylketonuria with a new medical food containing large neutral amino acids

(2017) D Concolino et al.   EUROPEAN JOURNAL OF CLINICAL NUTRITION

Neuropsychiatric comorbidities in adults with phenylketonuria: A retrospective cohort study

(2017) Deborah A. Bilder et al.   MOLECULAR GENETICS AND METABOLISM

The complete European guidelines on phenylketonuria: diagnosis and treatment

(2017) A. M. J. van Wegberg et al.   Orphanet Journal of Rare Diseases

Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients

(2017) Ania C. Muntau et al.   Orphanet Journal of Rare Diseases

Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With Phenylketonuria

(2016) Deborah A. Bilder et al.   DEVELOPMENTAL NEUROPSYCHOLOGY

Updated, web-based nutrition management guideline for PKU: An evidence and consensus based approach

(2016) Rani H. Singh et al.   MOLECULAR GENETICS AND METABOLISM

Large Neutral Amino Acid Supplementation Exerts Its Effect through Three Synergistic Mechanisms: Proof of Principle in Phenylketonuria Mice

(2015) Danique van Vliet et al.   PLoS One

Phenylalanine hydroxylase deficiency: diagnosis and management guideline

(2014)   GENETICS IN MEDICINE

Neurocognitive Evidence for Revision of Treatment Targets and Guidelines for Phenylketonuria

(2014) Rianne Jahja et al.   JOURNAL OF PEDIATRICS

Neurocognitive functioning in adults with phenylketonuria: Results of a long term study

(2013) J. Weglage et al.   MOLECULAR GENETICS AND METABOLISM

Suboptimal outcomes in patients with PKU treated early with diet alone: Revisiting the evidence

(2010) G.M. Enns et al.   MOLECULAR GENETICS AND METABOLISM

Improved nutritional management of phenylketonuria by using a diet containing glycomacropeptide compared with amino acids

(2009) Sandra C van Calcar et al.   AMERICAN JOURNAL OF CLINICAL NUTRITION

Cultural cognition of the risks and benefits of nanotechnology

(2008) Dan M. Kahan et al.   Nature Nanotechnology