Ornithine transcarbamylase deficiency Advocacy Hub | UniteRare

At a Glance

Live from database

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Regulatory Watchboard

10 events

Mar 2026Looking for VUS to Confirm Dominant Wolfram-like Syndrome Instead of Recessive Wolfram Syndrome

Hôpital Necker-Enfants Malades

TrialACTIVE NOT RECRUITING

Jan 2026Epiretinal Membrane in Patients With DR.

Assiut University

TrialNOT YET RECRUITING

Jul 2025Assessing Pharmacy Technician Educational Training for the Provision of Over-the-Counter Hearing Aids in Rural Alabama and Mississippi Pharmacies

University of Alabama, Tuscaloosa — NA

TrialRECRUITING

May 2025Role of OCT & OCT Angiography in Patients With Posterior at the Uveitis Clinic of Assiut University Hospital.

Assiut University

TrialNOT YET RECRUITING

Jan 2025Visual (Path)Ways in Multiple Sclerosis - Part II

University Hospital, Lille — NA

TrialRECRUITING

Dec 2024Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)

iECURE, Inc.

TrialRECRUITING

Nov 2024A Study to Evaluate the Pharmacodynamics and Safety of ARCT-810 in Participants With OTCD

Arcturus Therapeutics, Inc. — PHASE2

TrialRECRUITING

Apr 2024An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency

iECURE, Inc. — PHASE1, PHASE2

TrialRECRUITING

Nov 2023Halting Ornithine Transcarbamylase Deficiency With Recombinant AAV in ChildrEn

University College, London — PHASE1, PHASE2

TrialRECRUITING

Oct 2022Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency

Ultragenyx Pharmaceutical Inc — PHASE3

TrialACTIVE NOT RECRUITING

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

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

Total programs

Open now

Patient Assistance Programs1

Sodium Phenylbutyrate

GLENMARK PHARMACEUTICALS INC., USA

OpenContact for details

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Approved Treatments

2 FDA-approved

Sodium Phenylbutyrate

(SODIUM PHENYLBUTYRATE)Orphan drugstandard

GLENMARK PHARMACEUTICALS INC., USA

FDA label ↗

Sodium Phenylbutyrate

(SODIUM PHENYLBUTYRATE TABLETS, 500 MG)Orphan drugstandard

ENDO USA, Inc.

FDA label ↗

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Clinical Trial Landscape

8 active trials

1Phase 3

1Phase 2

4Unknown

2PHASE1, PHASE2

8Total recruiting

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Recent News & Research

1 article

Mol Ther Nucleic AcidsJun 16, 2026

The post-pandemic pivot: mRNA therapeutics enter the chronic rare disease arena

Published in Mol Ther Nucleic Acids. Giangrande PH et al.

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Specialist Network

Top 6 by expertise

Corina E Gonzalez, M.D.

National Cancer Institute (NCI)

📍 Bethesda, Maryland

Score 102 trials

Nicholas Ah Mew, MD

Children's National Research Institute

Score 75 trials

Peter J McGuire, M.D.

National Human Genome Research Institute (NHGRI)

Score 32 trials

Erin Rowell, MD

Ann & Robert H Lurie Children's Hospital of Chicago

📍 CHICAGO, IL

Score 32 trials

Berna Seker Yilmaz

Great Ormond Street Institute of Child Health

Score 05 pubs

Federicco Mingozzi

Genethon

Score 01 trial

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