Phenylketonuria
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CLINICAL_TRIALS trial_result · 2026-06-23Clinical Trial Update: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria [Phase 3]
Long-term safety study update for PTC923 in participants with Phenylketonuria (Phase 3).
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria [Phase 2]
This clinical trial update describes a Phase 2 study of JNT-517 in adolescents with phenylketonuria (PKU), sponsored by Otsuka Pharmaceutica…
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CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria [Phase 3]
This entry provides an update on a Phase 3 long-term safety study of PTC923 in people with Phenylketonuria, with the trial currently active …
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria [Phase 1]
A Phase 1 clinical trial to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of AG-181 in subjects with phenylketonuria, …
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria [Phase 3]
This clinical trial update describes a Phase 3 study to evaluate the long-term safety and efficacy of JNT-517 in participants with phenylket…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Effect 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 [NA]
This clinical trial examines how different meal types consumed before exercise affect plasma amino acids and metabolic control in people wit…
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CLINICAL_TRIALS other · 2026-04-13Clinical Trial Update: Evaluation of PKU Sphere in Italy [NA]
A clinical trial in Italy evaluating PKU Sphere for phenylketonuria has completed.
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CLINICAL_TRIALS trial_result · 2026-04-08Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU) [Phase 3]
Phase 3 study evaluating the safety and efficacy of JNT-517 in phenylketonuria is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq [Phase 4]
A Phase 4 clinical trial updates on rapid desensitization for adults with hypersensitivity reactions to Palynziq (pegvaliase) in phenylketon…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: A Study of Sepiapterin in Participants With Phenylketonuria (PKU) [Phase 3]
Phase 3 clinical trial investigating sepiapterin for phenylketonuria is currently recruiting participants.
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
This document outlines a long-term, post-marketing safety study of Palynziq in patients with phenylketonuria (PKU), and the study is current…
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
A long-term, post-marketing study to evaluate the immune response in patients treated with Palynziq for phenylketonuria, currently recruitin…
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Global multicenter clinical trial update evaluating maternal, fetal, and infant outcomes after exposure to Palynziq (pegvaliase) during preg…
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: Multiomics Approach in Adult Patients With Phenylketonuria
This entry describes a planned multiomics study in adult patients with phenylketonuria that is not yet recruiting.
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Immune Modulation During Palynziq® Treatment in Adults (IMPALA) [Phase 4]
A Phase 4 clinical trial update (IMPALA) evaluating immune modulation during Palynziq treatment in adults with phenylketonuria; the study is…
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CLINICAL_TRIALS trial_result · 2026-03-11Clinical Trial Update: Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU. [NA]
Terminated open-label, randomized 2-way crossover trial assessing daily phenylalanine fluctuation in phenylketonuria patients, comparing PKU…
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CLINICAL_TRIALS research · 2026-03-11Clinical Trial Update: Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers [NA]
The trial investigates the impact of elevated phenylalanine levels on brain function and cognition in adult carriers of phenylketonuria. Rec…
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: Safety and Efficacy Study of NGGT002 in PKU Adult Subjects [EARLY_Phase 1]
An early-phase clinical trial evaluating the safety and efficacy of NGGT002 in adults with phenylketonuria is currently recruiting.
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FDA approval · 2026-02-27FDA Approved: PALYNZIQ — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved BioMarin's supplemental application for PALYNZIQ (pegvaliase-pqpz), related to its regulatory action for phenylketonuria.
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria [EARLY_Phase 1]
A Phase 1 trial evaluated the efficacy and safety of orally administered engineered probiotic CBT102-A in children with phenylketonuria; the…
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: Evaluation of PKU UP [NA]
A clinical trial update for PKU UP, sponsored by Vitaflo International, Ltd, indicates the study in phenylketonuria is active but not recrui…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: The Psychosocial Functioning of Adults With Phenylketonuria.
Clinical trial update on the psychosocial functioning of adults with phenylketonuria, sponsored by Central Hospital in Nancy, France; status…
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EMA approval · 2026-02-11EMA Authorised: Sephience (sepiapterin) — PTC Therapeutics International Limited
The EMA authorised Sephience (sepiapterin) for phenylketonuria, with orphan medicine status, and PTC Therapeutics International Limited as t…
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EMA approval · 2026-02-03EMA Authorised: Sapropterin Dipharma (sapropterin dihydrochloride) — Dipharma Arzneimittel GmbH
The EMA authorised Sapropterin Dipharma (sapropterin dihydrochloride) for phenylketonuria, with Dipharma Arzneimittel GmbH as the marketing …
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: PKU Protein Requirements [NA]
Emory University is conducting a clinical trial to study protein requirements in phenylketonuria, but recruitment has not yet started.