UCDC > About Us
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`https://www.rarediseasesnetwork.org/cms/ucdc/About-Us
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`This consortium is part of RDCRN , an initiative of ORDR, NCATS
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`About Us
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`About UCDC
`
`The Urea Cycle Disorders Consortium (UCDC) consists of a team of physician scientists, neuropsychologists, nurses, genetic
`counselors, research staff, and patient advocates throughout the US, working together to improve the lives of people with urea cycle
`disorders.
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`The UCDC is an international collaborative Consortium with sites in United States, Canada, and Europe. The sites have significant
`experience in the diagnosis and treatment as well as research of urea cycle disorders and are working together to improve the lives
`of people with urea cycle disorders.
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`As urea cycle disorders are rare, the number of participants that can be enrolled is limited and thus, the conduct of clinical research
`studies is very challenging. The Consortium provides a way for patients to join with doctors and researchers by participating in
`research studies. The greater the collaboration between researchers and patients, the more we can learn about these disorders.
`This is an important step in understanding how these diseases impact the lives of affected individuals and discover new and
`improved treatments.
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`Mission
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`UCDC Leadership
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`The UCDC is dedicated to improving the lives of individuals affected by
`urea cycle disorders. The mission of the UCDC is to understand the natural
`history of the urea cycle disorders, conduct clinical research and clinical
`trials to discover new treatments, form partnership with patients, patient
`advocacy groups, and other stakeholders and train the next generation of
`rare disease investigators.
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`We hope to accomplish our goals by:
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`Continuing the enrollment of a large group of patients with urea cycle
`disorders into an observational study called the Longitudinal Study of
`Urea Cycle Disorders. In this study, subjects are followed over time
`and clinical, neuropsychological, laboratory and other data are
`collected at regular intervals.
`
`Mendel Tuchman, MD
`Children's National Medical Center, Washington,
`D.C.
`Principal Investigator
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`Shawn McCandless, MD
`Rainbow Babies & Children’s Hospital/Case Western
`Reserve University, Cleveland, OH
`Co-Principal Investigator, Administrative Director
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`Cynthia LeMons
`Executive Director, National Urea Cycle Disorders
`Foundation
`Co-Principal Investigator
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`Horizon Exhibit 2027
`Lupin v. Horizon
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`UCDC > About Us
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`https://www.rarediseasesnetwork.org/cms/ucdc/About-Us
`
`Sandesh CS Nagamani, MD
`Baylor College of Medicine, Houston, TX
`Co-Principal Investigator
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`Conducting clinical trials of promising new therapies for the treatment
`of urea cycle disorders.
`Establishing a patient contact registry.
`Discovering new laboratory markers of disease that will lead to better
`treatment and deeper scientific understanding of the causes of these
`diseases.
`Working with the National Urea Cycle Disorders Foundation to help
`patients who wish to be involved in research connect with doctors
`conducting the research.
`Constructing and maintaining an electronic website resource with
`updated information for clinicians, researchers, and patients.
`Mentoring and training junior investigators in UCD and rare diseases
`research.
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`Goals
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`The UCDC works to achieve its mission through four major goals:
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`Further the understanding of the natural history of urea cycle disorders1.
`
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`Develop and conduct clinical research and clinical trials to discover2.
`new therapies that improve survival and outcome
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`Create resources with information on urea cycle disorders for3.
`clinicians, researchers, and patients
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`Train the next generation of rare disease investigators, particularly in4.
`the field of UCD
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