Research Studies
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(argininosuccinic aciduria, ASL deficiency)
UCD Consortium NIH Office of Rare Diseases-sponsored Research Studies
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Longitudinal Study for UCDswith list of all UCD Consortium Research Centers
Completed Studies
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Assessing Neural Mechanisms of injury in inborn errors of urea metabolism using Structural MRI, Functional MRI, and Magnetic Resonance Spectroscopy
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Nitric Oxide Flux and Ureagenesis in Argininosuccinate Synthetase Deficiency (ASSD) (Citrullinemia I)
Industry (Pharmaceutical Company) Sponsored Clinical Trials
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Safety, Tolerability, and Pharmacokinetics of ARCT-810 in Stable Adult Subjects With Ornithine Transcarbamylase Deficiency- Recruiting
(Sponsor: Arcturus Therapeutics) -
About mRNA and ARCT-810 as an investigational therapy for OTCFor more information or help connecting with a trial site, contact NUCDF
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CAEB1102-300A is a multi-center randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of pegzilarginase in patients with ARG1-D Active, not recruiting
(Sponsor: Aeglea Biotherapeutics)
If you have questions or would like to discuss any of the studies or clinical trials, please contact NUCDF at info@nucdf.org
“I have been a participant or observer in many efforts to bring together families and researchers in regard to a specific disorder or group of disorders, and I have never seen one in which there was such a superb collaboration and focus on the common goal.”
Hugo Moser, M.D., Adrenoleukodystrophy Researcher (“Lorenzo’s Oil”), Kennedy-Krieger Institute, NIH Monitor to UCDC
