Kuvan® in Phenylketonuria Patients Less Than 4 Years Old

Phenylketonuria

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3) The Effect of Nursing Empowerment Program Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute. Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria Impact of Fruit and Vegetables in PKU Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency Additional Dietary Large Neutral Amino Acids (LNAA) for Improved Symptoms in Adult Classical Phenylktonuria (PKU) A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference? Evaluation of PKU Sphere Liquid A Study of PTC923 in Participants With Phenylketonuria Evaluation of PKU Express Plus Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients Remote Neurocognitive and Psychological Assessment in PKU Evaluation of PKU Explore France Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria Natural History Clinical Study in Adult PKU Evaluation of PKU Sphere in Italy Efficacy and Safety of SYNB1618 in Adult Patients With Phenylketonuria AAV Gene Therapy Study for Subjects With PKU A Prospective Clinical Study of Phenylketonuria (PKU) Life With Phenylketonuria. Adult Neurological Outcome of PCU Screened Patients From 1971 to 2002. Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU) Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism Bone Mineral Density in Adults With Hyperphenylalaninemia Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy The Early History of Universal Screening for Metabolic Disorders Market Research – Acceptability Study for a New PKU Protein Substitute Market Research – Acceptability Trial for a New PKU Amino Acid Based Protein Substitute Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders The Effectiveness of Kuvan in Amish PKU Patients An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) Study of BH4, a New and Simple Treatment of Mild PKU Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia Testing of Four Home Phenylalanine Monitoring Prototype Devices Evaluation of PKU Start Evaluation of PKU Explore A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects The Effects of CGMP in Children and Adults With PKU Nutrition Status of Adults With PKU Before and During Treatment With Pegvaliase SNAP: Study Nutrients in Adult PKU Effects of Kuvan on Melatonin Secretion A Pilot Study on Diurnal Variation PANDA: PKU Amino Acid Evaluation A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients Sapropterin Expanded Access Program Study to Evaluate the Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU PKU Skin Stripping A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302 Gene Therapy Clinical Study in Adult PKU Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet Follow-up of Adult Phenylketonuria (PKU) Patients PKU Low Calorie Drink Study A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKU Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks Nutrition Status of Adults Treated With Pegvaliase Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria An Observational, Cross-sectional Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD). GMP Drink for PKU Study Nutritional and Neurotransmitter Changes in PKU Subjects on BH4 Evaluation of PKU Sphere Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years – Study on Phenylalanine Tolerance and Safety Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With Phenylketonuria Safety and Tolerability of RTX-134 in Adults With Phenylketonuria Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU) Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) Phenylalanine and Its Impact on Cognition A Non-Interventional Interview Study of Phenylketonuria (PKU) Adults Long-Term Follow Up Study of Subjects Previously Administered HMI 102 Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry The Effect of Supplemental Docosahexaenoic Acid (DHA) on Neurocognitive Outcomes in Teen and Adult Women With Phenylketonuria(PKU) Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients Simple Breath Test to Examine Phenylalanine Metabolism MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria Body Composition, Energy Intake and Expenditure in People With Phenylketonuria Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period Simplified Diet Approach in Phenylketonuria Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU) Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With Phenylketonuria Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated Phenylketonuria The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU) The Brain, Neurological Features and Neuropsychological Functioning in Adults With Phenylketonuria: A Pilot Study Protein Requirements in Adults With Phenylketonuria (PKU) Moderate Intensity Exercise and Phenylketonuria Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of Phenylketonuria Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive Behaviors Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006 Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy Protein Requirements in Children With Phenylketonuria (PKU) Kuvan®’s Effect on the Cognition of Children With Phenylketonuria Phenylketonuria, Oxidative Stress, and BH4 Quantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With Phenylketonuria Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia Kuvan® in Phenylketonuria Patients Less Than 4 Years Old BH4 Responsiveness in PAH Deficiency PKU Patients Response to Phenylketonuria to Tetrahydrobiopterin (BH4) Behavioral Effects of Kuvan in Children With Mild Phenylketonuria Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced Thermogenesis Nutritional Status in Phenylketonuria Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development. Liver Cell Transplant for Phenylketonuria Sapropterin in Individuals With Phenylketonuria Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria Observational Study of Endothelial Dysfunction in Phenylketonuria