Records containing: Phenylketonuria
This is the wording recorded or indexed by the source. An agency index term is not necessarily an authorised indication. Co-listed drug names are not linked to this term as a treatment relationship.
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CLINICAL_TRIALS study record update · 2026-09-09A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
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CLINICAL_TRIALS study record update · 2026-06-23A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
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CLINICAL_TRIALS study record update · 2026-04-24A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
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CLINICAL_TRIALS study record update · 2026-04-24A Phase 1b, Open-label, Multicenter, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of AG-181 in Subjects With Phenylketonuria
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CLINICAL_TRIALS study record update · 2026-04-16Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises
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CLINICAL_TRIALS study record update · 2026-04-08A Phase 3, Double-Blind, Randomized, Two-Period, Multicenter, Placebo-Controlled, Efficacy and Safety Study of JNT-517 for the Treatment of Participants With Phenylketonuria
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CLINICAL_TRIALS study record update · 2026-04-03A Phase 4 Study to Evaluate the Impact of a Rapid Drug Desensitization (RDD) Protocol for Adults With Phenylketonuria and Experiencing Hypersensitivity Reactions to Palynziq
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CLINICAL_TRIALS study record update · 2026-03-27A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
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CLINICAL_TRIALS study record update · 2026-03-17A Phase 4 Study of Immune Modulation During Palynziq® Treatment in Adults With Phenylketonuria (PKU)
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CLINICAL_TRIALS study record update · 2026-02-27Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria：a Randomized, Double-blind, Placebo-controlled, Parallel-group Clinical Study
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CLINICAL_TRIALS study record update · 2025-12-22Etude de l'INflammation systémique de Bas GRade Chez Les Patients Adultes Atteints de PHénylcétonurie
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CLINICAL_TRIALS study record update · 2025-12-19Evaluating the Adherence, Tolerance, Acceptability and Safety of New GMP-based Powdered Protein Substitutes in PKU and TYR: a Case Study Series
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CLINICAL_TRIALS study record update · 2025-09-23A Phase 1/Phase 2, Open-label, Dose-escalation, and Dose Expansion Study to Evaluate the Safety, Tolerability, and Efficacy of SAR444836, an Adeno-associated Viral Vector-mediated Gene Transfer of Human Phenylalanine Hydroxylase, in Adult Participants With Phenylketonuria
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CLINICAL_TRIALS study record update · 2025-07-31A Phase 1/2, First-In-Human, Multiple Part, Single Ascending and Multiple Dose Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria
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CLINICAL_TRIALS study record update · 2025-04-04Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria Two Phases Study 1- Cross Sectional Comparative Study ( PKU Versus Non PKU Infants and Children). 2- Open Label Randomized Controlled Clinical Trial for PKU Patients Only. 1/ Intervention Group: 44 Patients Will Treated With L-carnitine at a Dose of 100 mg/kg/Dose for One Year Beside Their Treatment. 2/ Control PKU Group: 44 Patients Will Not Receive L-carnitine Supplementation Beside Their Treatment 3/ Control Healthy Group Same Age and Sex
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CLINICAL_TRIALS study record update · 2025-04-03Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria (PKU) or Maple Syrup Urine Disease (MSUD).
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CLINICAL_TRIALS study record update · 2024-10-22A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of mRNA 3210 in Participants With Phenylketonuria
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CLINICAL_TRIALS study record update · 2024-07-25Protein Requirements in Phenylketonuria (PKU) Patients Compared Using PKU Sphere™ | Glycomacropeptide (GMP) and an L-amino Acid-based Product
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CLINICAL_TRIALS study record update · 2024-06-20A Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3)
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CLINICAL_TRIALS study record update · 2024-03-01Announcement of Rare Metabolic Diseases as Part of Systematic New-born Screening: the Experience of Phenylketonuria.
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CLINICAL_TRIALS study record update · 2024-02-16A Study to Evaluate the Acceptability of PKU Explore, a Renovated Phenylalanine Free Second Stage Protein Substitute for Use in the Dietary Management of Phenylketonuria in Infants From 6 Months to 5 Years of Age With Regard to Product Tolerance and Adherence.
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CLINICAL_TRIALS study record update · 2024-02-16A Study to Evaluate the Acceptability of a New Phenylalanine Free Infant Formula for Use in the Dietary Management of Phenylketonuria in Infants From Birth to 2 Year of Age With Regard to Product Tolerance and Adherence.
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CLINICAL_TRIALS study record update · 2024-01-10A Phase 3 Study of PTC923 in Subjects With Phenylketonuria
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CLINICAL_TRIALS study record update · 2023-12-22Evaluation of Capillary Blood Collection Devices (Mitra® and HemaPEN®) in the Context of Metabolic Diseases and, in Particular, Pediatric Monitoring of Phenylketonuria
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CLINICAL_TRIALS study record update · 2023-10-10A Phase 1, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of HMI-103 Administered Intravenously in Adult Participants With Classical PKU Due to PAH Deficiency