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Topics

Learn about phenylketonuria topics

Real World Evidence

Poster

Topics

Real World Evidence

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Real-world safety and tolerability of pegvaliase: A non-interventional surveillance study in Japan

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Initial psychometric evaluation of the Adult Symptom Severity and Impacts Scale (PKU-SSIS) using an interim data cut from the OPAL study

Occurrence of anaphylaxis in adult incident pegvaliase-treated PKU patients in a post-marketing safety analysis in the United States

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Lessons learned from 5 years of experience with pegvaliase in US clinics: a case series

Lessons learned from 5 years of experience with pegvaliase in US clinics: a case series

Reduction of blood phenylalanine in participants enrolled in OPAL, an observational study, mirror findings from the US-based PRISM population

OPAL: A multicenter, observational study to evaluate the real-world outcomes of pegvaliase in adults with phenylketonuria

Kuvan® Adult Maternal Paediatric European Registry (KAMPER): Final results in phenylketonuria (PKU) patients

Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU-MOMs sub-registries

Safety and Efficacy Profile

Management of pegvaliase-related skin concerns: best practice recommendations using a modified Delphi approach

Index of sustained Phe response and improvements in PKU clinical outcome assessments in patients receiving pegvaliase

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Safety and efficacy of pegvaliase in adolescents with phenylketonuria: Primary results from PEGASUS, a phase 3 open-label randomized controlled study

PALLADIUM: A phase 4 study to evaluate a rapid drug desensitization protocol for adults with phenylketonuria experiencing hypersensitivity reactions to pegvaliase

Real-world safety and tolerability of pegvaliase: A non-interventional surveillance study in Japan

Long-term management strategies for pegvaliase use in phenylketonuria: Lessons learned from the phase-3 PRISM open-label extension study

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Index of sustained Phe response and improvements in PKU clinical outcome assessments in patients receiving pegvaliase

Index of sustained Phe response and improvements in PKU clinical outcome assessments in patients receiving pegvaliase

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Improvements in blood phenylalanine and health-related quality of life outcomes among adults with PKU receiving pegvaliase in the OPAL study

Occurrence of anaphylaxis in adult incident pegvaliase-treated PKU patients in a post-marketing safety analysis in the United States

Initial psychometric evaluation of the Adult Symptom Severity and Impacts Scale (PKU-SSIS) using an interim data cut from the OPAL study

Index of sustained Phe response and improvements in PKU clinical outcome assessments in patients receiving pegvaliase

Final safety and efficacy of pegvaliase in Japanese adults with phenylketonuria

Lessons learned from 5 years of experience with pegvaliase in US clinics: a case series

Lessons learned from 5 years of experience with pegvaliase in US clinics: a case series

Reduction of blood phenylalanine in participants enrolled in OPAL, an observational study, mirror findings from the US-based PRISM population

Three-year interim safety and efficacy of pegvaliase in Japanese adults with phenylketonuria

The long-term safety and efficacy of pegvaliase 60 mg/day in adults with phenylketonuria

Two-year Interim Safety and Efficacy of pegvaliase in Japanese adults with Phenylketonuria

The long-term safety and efficacy of pegvaliase 60 mg/day in adults with Phenylketonuria

Kuvan® Adult Maternal Paediatric European Registry (KAMPER): Final results in phenylketonuria (PKU) patients

Efficacy and safety of sapropterin before and during pregnancy: Final analysis of the Kuvan® Adult Maternal Paediatric European Registry (KAMPER) and Phenylketonuria Developmental Outcomes and Safety (PKUDOS) PKU-MOMs sub-registries

Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies

Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies

Efficacy and safety of the recommended pegvaliase dosing regimen in adults with phenylketonuria in the phase 3 PRISM studies

ENDURE: A phase IV, open-label trial to assess the responsiveness of patients with phenylketonuria in Denmark to treatment with sapropterin over 28 days

Study 165-305: Interim Safety and Efficacy of Pegvaliase in Japanese Adults with Phenylketonuria

Efficacy and Safety of the recommended Pegvaliase dosing regimen in adults with Phenylketonuria in the Phase 3 Prism Studies

Phase 3 PRISM studies: Efficacy and safety of pegvaliase in patients aged 16 and 17 years with phenylketonuria

Phase 3 PRISM studies: Update of efficacy and safety of pegvaliase for the treatment of adults with phenylketonuria

Phase 3 PRISM studies: Efficacy and safety of pegvaliase 60 mg dose in adult patients with phenylketonuria

Evaluating change in diet with pegvaliase treatment in adults with phenylketonuria: Results from phase 2 and 3 clinical trials

Effect of sapropterin dihydrochloride on IQ preservation in children (aged 0–6 years) with phenylketonuria

Dietary intakes and adverse events in pegvaliase-treated phenylketonuria adults who had low blood phenylalanine concentrations

Phase 3 PRISM studies: Efficacy & safety of pegvaliase 60mg in adult patients with PKU

Effect Of Sapropterin Dihydrochloride On IQ Preservation In Children (Aged 0-6 Years) With Phenylketonuria

Phase 3 PRISM studies: Efficacy and safety of pegvaliase 60 mg dose in adult patients with phenylketonuria

Phase 3 PRISM studies: Efficacy and safety of pegvaliase in patients 16-17 years of age with phenylketonuria

Burden of Disease

Neuropsychiatric comorbidities in adolescents with PKU in the United States

Neuropsychiatric comorbidities in adolescents with PKU in the United States

Work ability in adults with PKU in Sweden in 2020

Neuropsychiatric comorbidities in adolescents with PKU in the United States

Lifetime monitoring of Phe levels in PKU from birth to adulthood in the Swedish Registry for Inherited Metabolic Diseases

Neuropsychiatric comorbidities in adults with PKU in Sweden

European guidelines on diagnosis and treatment of phenylketonuria: First revision

Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG)

PKU: Burden of Illness Dr. Deborah Bilder | University of Utah

Assessment of the Treatment and Management LAndScape of Phenylketonuria: ATLAS Survey Study in Japan

Evaluating trends in metabolic control and treatment history among pegvaliase trial participants

Systematic literature review of prevalence and severity of somatic comorbidities in adults with PKU

Evaluating trends in self-rated historic metabolic control and treatment history among PRISM participants

Evaluating trends in self-rated historic metabolic control and treatment history among PRISM participants

Assessing the health status of ≥16-year-old patients with early-diagnosed PKU from the retrospective analysis of the full French insurance claims cohort 2006-2018

Assessing the health status of patients with late-diagnosed PKU from the retrospective analysis of the full French insurance claims cohort 2006-2018

Measuring phenylketonuria burden of illness: Development of the Phenylketonuria Symptom Severity and Impacts Scale as a robust patient reported outcome

Birth prevalence of phenylalanine hydroxylase deficiency: A systematic literature review and meta-analysis

Measuring phenylketonuria burden of illness: Development of the Phenylketonuria Symptom Severity and Impacts Scale as a robust patient reported outcome

Neurocognitive, neurological and neuropsychological manifestations of Phenylketonuria (PKU)

Evaluating trends in self-rated historic metabolic control and treatment history among PRISM participants

Systematic literature review of the prevalence and severity of somatic comorbidities in adults with phenylketonuria

Kuvan^® Adult Maternal Paediatric European Registry (KAMPER): Final results in phenylketonuria (PKU) patients

Kuvan^® Adult Maternal Paediatric European Registry (KAMPER): Final results in phenylketonuria (PKU) patients

PKU: Burden of Illness
Dr. Deborah Bilder | University of Utah