Disorder of phenylalanine metabolism Advocacy Hub | UniteRare

At a Glance

Live from database

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4Recruiting trials

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23Specialists

Regulatory Watchboard

9 events

Mar 2026Multiomics Approach in Adult Patients With Phenylketonuria

University Hospital, Tours

TrialNOT YET RECRUITING

Jan 2026The Psychosocial Functioning of Adults With Phenylketonuria.

Central Hospital, Nancy, France

TrialNOT YET RECRUITING

Jan 2026Assessment of Gut Microbiota-Derived Amino Acid Metabolite Production in Patients With MASLD

Hospices Civils de Lyon — NA

TrialNOT YET RECRUITING

Dec 2025To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

Gritgen Therapeutics Co., Ltd. — EARLY_PHASE1

TrialNOT YET RECRUITING

Dec 2025Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers

University of Missouri-Columbia — NA

TrialRECRUITING

Jul 2025A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria

Otsuka Pharmaceutical Development & Commercialization, Inc. — PHASE2

TrialRECRUITING

Jun 2025A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria

Otsuka Pharmaceutical Development & Commercialization, Inc. — PHASE3

TrialNOT YET RECRUITING

Nov 2022A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding

BioMarin Pharmaceutical — NA

TrialRECRUITING

Sep 2020AAV Gene Therapy Study for Subjects with PKU

BioMarin Pharmaceutical — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

FINANCIAL LANDSCAPE SUMMARY

Total programs

No financial assistance programs currently listed for Disorder of phenylalanine metabolism.
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Clinical Trial Landscape

4 active trials

1Phase 2

2N/A

1PHASE1, PHASE2

4Total recruiting

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Recent News & Research

No recent news articles indexed yet for Disorder of phenylalanine metabolism.
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Specialist Network

Top 6 by expertise

Minjoo Kim, MS, DO

Yonsei University

📍 PALMETTO, FL

Score 61 trial

Joshua Lilienstein, M.D., MD

BioMarin Pharmaceutical

📍 JAMAICA PLAIN, MA

Score 61 trial

Rajavel Elango, PhD

BC Children's Hospital Research Institute, University of British Columbia

📍 Vancouver, British Columbia

Score 65 trials

Annette Feigenbaum, MD, M.D

The Hospital for Sick Children

📍 SAN DIEGO, CA

Score 51 trial1 pub

Louis J Elsas, MD

University of Miami

Score 41 trial1 pub

Raed Selmi, MD-PhD

ASST Santi Paolo e Carlo

Score 41 trial

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