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

[1]  J. Vockley,et al.  Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial. , 2018, Molecular genetics and metabolism.

[2]  Janet A. Thomas,et al.  Strategies for Successful Long-Term Engagement of Adults With Phenylalanine Hydroxylase Deficiency Returning to the Clinic: , 2017 .

[3]  P. Fitzpatrick,et al.  Formulation and PEGylation optimization of the therapeutic PEGylated phenylalanine ammonia lyase for the treatment of phenylketonuria , 2017, PloS one.

[4]  C. Romani,et al.  The Impact of Phenylalanine Levels on Cognitive Outcomes in Adults With Phenylketonuria: Effects Across Tasks and Developmental Stages , 2017, Neuropsychology.

[5]  S. Cederbaum,et al.  Adherence to clinic recommendations among patients with phenylketonuria in the United States. , 2017, Molecular genetics and metabolism.

[6]  C. Romani,et al.  Cognitive Outcomes in Early-Treated Adults With Phenylketonuria (PKU): A Comprehensive Picture Across Domains , 2017, Neuropsychology.

[7]  K. Wyrwich,et al.  An Adaptation of the Profile of Mood States for Use in Adults With Phenylketonuria , 2016 .

[8]  W. Irish,et al.  Systematic Review and Meta-Analysis of Neuropsychiatric Symptoms and Executive Functioning in Adults With Phenylketonuria , 2016, Developmental neuropsychology.

[9]  S. Nadler,et al.  Immunogenicity to Biotherapeutics – The Role of Anti-drug Immune Complexes , 2016, Front. Immunol..

[10]  U. Lichter-Konecki,et al.  Phenylketonuria (PKU): A problem solved? , 2015, Molecular genetics and metabolism reports.

[11]  E. Bettiol,et al.  Assessment of the impact of phenylketonuria and its treatment on quality of life of patients and parents from seven European countries , 2015, Orphanet Journal of Rare Diseases.

[12]  K. Wyrwich,et al.  Evaluation of Neuropsychiatric Function in Phenylketonuria: Psychometric Properties of the ADHD Rating Scale-IV and Adult ADHD Self-Report Scale Inattention Subscale in Phenylketonuria. , 2015, Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research.

[13]  S. Stahl,et al.  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. , 2015, Molecular genetics and metabolism.

[14]  T. Anchordoquy,et al.  Potential induction of anti-PEG antibodies and complement activation toward PEGylated therapeutics. , 2014, Drug discovery today.

[15]  P. Strisciuglio,et al.  New Strategies for the Treatment of Phenylketonuria (PKU) , 2014, Metabolites.

[16]  J. Vockley,et al.  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 , 2014, The Lancet.

[17]  H. Eisen Affinity Enhancement of Antibodies: How Low-Affinity Antibodies Produced Early in Immune Responses Are Followed by High-Affinity Antibodies Later and in Memory B-Cell Responses , 2014, Cancer Immunology Research.

[18]  John J. Mitchell,et al.  Phenylalanine hydroxylase deficiency: diagnosis and management guideline , 2013, Genetics in Medicine.

[19]  S. Cederbaum,et al.  Newborn screening 50 years later: access issues faced by adults with PKU , 2013, Genetics in Medicine.

[20]  C. Hollak,et al.  High phenylalanine levels directly affect mood and sustained attention in adults with phenylketonuria: a randomised, double-blind, placebo-controlled, crossover trial , 2010, Journal of Inherited Metabolic Disease.

[21]  P. Burgard,et al.  The reality of dietary compliance in the management of phenylketonuria , 2010, Journal of Inherited Metabolic Disease.

[22]  J. Vockley,et al.  Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study. , 2009, The Journal of pediatrics.

[23]  M. Bik-Multanowski,et al.  Quality of life in noncompliant adults with phenylketonuria after resumption of the diet , 2008, Journal of Inherited Metabolic Disease.

[24]  S. Cederbaum,et al.  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 study , 2007, Journal of Inherited Metabolic Disease.

[25]  A. Chakrapani,et al.  Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study , 2007, The Lancet.

[26]  D. Hepner,et al.  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. , 2006, The Journal of allergy and clinical immunology.

[27]  S. Brown Clinical features and severity grading of anaphylaxis. , 2004, The Journal of allergy and clinical immunology.

[28]  D. Chaplin Overview of the immune response. , 2003, The Journal of allergy and clinical immunology.

[29]  B. Burton,et al.  Phenylketonuria in adulthood: A collaborative study , 2002, Journal of Inherited Metabolic Disease.

[30]  A. Boneh,et al.  How practical are recommendations for dietary control in phenylketonuria? , 2002, The Lancet.

[31]  T. Chang,et al.  A different approach to treatment of phenylketonuria: phenylalanine degradation with recombinant phenylalanine ammonia lyase. , 1999, Proceedings of the National Academy of Sciences of the United States of America.

[32]  K. Wyrwich,et al.  Content Validity of the ADHD Rating Scale (ADHD RS-IV) and Adult ADHD Self-Report Scale (ASRS) in Phenylketonuria: , 2016 .

[33]  J. Walter,et al.  Follow up of phenylketonuria patients. , 2011, Molecular genetics and metabolism.

[34]  Wen-chao Song,et al.  Complement and its role in innate and adaptive immune responses , 2010, Cell Research.