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Results for “Amino-Acid Transport Disorder”

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Showing 7 of 0 results

Very early researchStudy completedNCT01659645
What this trial is testing

Anti-1-amino-3-[18F]Fluorocyclobutyl-1-carboxylic Acid (Anti-[18F](FACBC)Positron Emission Tomography (PET-CT) of the Breast

Who this might be right for
Breast Cancer
Emory University 12
Early research (Phase 1)Looking for participantsNCT07643844
What this trial is testing

AAVrh10-PCCA Gene Therapy for Propionic Acidemia

Who this might be right for
Propionic Acidemia
Mayo Clinic 9
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Testing effectiveness (Phase 2)Looking for participantsNCT07667387
What this trial is testing

Study of LNP.UCD.ABE in Patients With Urea Cycle Disorders

Who this might be right for
Urea Cycle DisordersCarbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas 7
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07410455
What this trial is testing

An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome

Who this might be right for
Lowe Syndrome
Can-Fite BioPharma 5
Testing effectiveness (Phase 2)Study completedNCT02991144
What this trial is testing

Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset Ornithine Transcarbamylase (OTC) Deficiency

Who this might be right for
Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc 16
Not applicableLooking for participantsNCT02780297
What this trial is testing

Prospective Research Rare Kidney Stones (ProRKS)

Who this might be right for
HyperoxaluriaCystinuriaDent Disease+2 more
Mayo Clinic 220