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Clinical Trials for Phenylketonurias

• A Dose-finding Study to Evaluate mRNA-3210 in Participants With 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)
• Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
• Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency
• A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
• A Study of PTC923 in Participants With Phenylketonuria
• Efficacy and Safety of SYNB1618 in Adult Patients With Phenylketonuria
• AAV Gene Therapy Study for Subjects With PKU
• Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU Patients
• BH4 Responsiveness in PAH Deficiency PKU Patients
• Safety and Tolerability of RTX-134 in Adults With Phenylketonuria
• Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)
• O2 Transport and Utilization in Health and Lung Disease
• Phenylalanine and Its Impact on Cognition
• Study to Evaluate the Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
• A Study of CNSA-001 in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
• Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria
• The Effectiveness of Kuvan in Amish PKU Patients
• Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria
• Kuvan®'s Effect on the Cognition of Children With Phenylketonuria
• Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU
• An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
• A Pilot Study on Diurnal Variation
• Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria
• Effects of Kuvan on Melatonin Secretion
• Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy
• Liver Cell Transplant for Phenylketonuria
• Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in Phenylketonuria
• Phenylketonuria, Oxidative Stress, and BH4
• Kuvan® in Phenylketonuria Patients Less Than 4 Years Old
• Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan Nonresponders
• Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)
• Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
• Response to Kuvan® in Subjects With Phenylketonuria (PKU) in a 4 Weeks Testing Period
• Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy
• The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)
• 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)
• Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
• Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU
• Behavioral Effects of Kuvan in Children With Mild Phenylketonuria
• A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects
• PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
• Sapropterin in Individuals With Phenylketonuria
• Nutritional and Neurotransmitter Changes in PKU Subjects on BH4
• Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria
• Sapropterin Expanded Access Program
• Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and Safety
• Safety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 Deficiency
• Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
• Study of Phenoptin to Increase Phenylalanine Tolerance in Phenylketonuric Children on a Phenylalanine-restricted Diet
• Study of BH4, a New and Simple Treatment of Mild PKU
• Response to Phenylketonuria to Tetrahydrobiopterin (BH4)
• A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine Levels
• Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With Phenylketonuria
• Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels