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Health / Urea cycle disorder / Ornithine transcarbamylase deficiency / Argininosuccinic aciduria / Hyperammonemia / Ornithine translocase deficiency / Arginase / Citrullinemia / N-Acetylglutamate synthase deficiency / Urea cycle / Metabolism / Biology


UCDC Newsletter_4.22.13_Final.pub
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Document Date: 2013-04-23 17:35:32


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File Size: 1,18 MB

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City

York / Philadelphia / Zurich / /

Company

aciduria ASA / Children / /

Country

Switzerland / United States / /

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Event

FDA Phase / /

Facility

Colorado University of Heidelberg Heidelberg / Science University / Pennsylvania University of Minnesota Amplatz Children’s Hospital Minneapolis / Children’s Hospital / Case Western Reserve University / California The Hospital / Washington Baylor College of Medicine Houston / Germany Children’s Hospital of Philadelphia Philadelphia / Children’s Hospital of Philadelphia University of California at Los Angeles Mount Sinai School / Participating Centers Children’s National Medical Center / Cleveland Hospital / Sciences University / Portland The Children's Hospital / DC Seattle Children’s Hospital / New York Children’s National Medical Center / UCD Subtype Currently On Study Baylor College of Medicine / Switzerland The Children’s Hospital / Toronto University Children’s Hospital / New York Boston Children’s Hospital / Zurich Seattle Children’s Hospital / University of Minnesota University / Texas University Children’s Hospital / /

IndustryTerm

routine magnetic resonance imaging / neural networks / diffusion tensor imaging / imaging / clinical site / /

MarketIndex

OTC deficiency were transplanted on / /

MedicalCondition

rare diseases / Total ASLD Arginosuccinate lyase deficiency / Urea Cycle Disorders / Seizures / Brain Injury / Partial Ornithine Transcarbamylase Deficiency / neonatal onset citrullinemia / dysfunction / citrullinemia / OTC deficiency / injury / disorder / neonatal onset OTC deficiency / argininosuccinic acid synthetase deficiency / ADHD / late-onset disease / chronic treatment / CPS1 deficiency / infection / Inborn Errors of Metabolism / ASA ASSD Argininosuccinate synthetase deficiency / hepatocellular injury / disorders / liver dysfunction / liver failure / respiratory tract infections / infectious precipitants / MS / infections / acute liver failure / disease / rare disease / ornithine deficiency Citrulline deficiency / Inflammation / neonatal onset disease / hyperammonemia / homocitrullinuria syndrome / /

MedicalTreatment

liver transplants / /

Organization

New York Children’s National Medical Center / National Urea Cycle Disorders Foundation / Hospital for Sick Children Toronto / Canada Mount Sinai School / Children’s Hospital Aurora / Washington Baylor College of Medicine Houston / Case Western Reserve University / New York Boston Children’s Hospital Boston / Texas University Children’s Hospital Zurich / University of Minnesota University / DC Seattle Children’s Hospital Seattle / Rotenberg Family Fund / Children’s National Medical Center Office / School of Medicine / Hospital Boston / Massachusetts Oregon Health and Science University Portland / Colorado University of Heidelberg Heidelberg / Dietmar-Hopp Foundation / FDA / Children's Hospital / University of California at Los Angeles / UCLA / Hospital Oregon Health and Sciences University / National UCD Foundation / O’Malley Foundation / Yale / Urea Cycle Disorders Consortium / Cleveland Hospital for Sick Children / Children’s Hospital / Pennsylvania University of Minnesota Amplatz Children’s Hospital Minneapolis / Science University Portland / Oregon Rainbow Babies and Children’s Hospital Cleveland / Baylor College of Medicine / Houston / DC Rainbow Babies and Children’s Hospital / Toronto University Children’s Hospital / UCD / Kettering Fund / /

Person

Brendan Lee / Peter McGuire / Philippe Campeau / Renata Gallagher / Sodium Phenylbutyrate (Buphenyl) / Mark Batshaw / Tamar Stricker / Mendel Tuchman / Jennifer Seminara / Andrea Gropman / Ursina Spörri / Identifies Symptomatic / Matthias Baumgartner / Sandesh C. Sreenath Nagamani / Marshall Summar / Fabienne Dietrich / Glycerol Phenylbutyrate / Lee B. Mol Genet Metab / Brian Smith / Oleg Shchelochkov / Cynthia Le Mons / /

/

Position

Executive Director / study coordinator / physician / Director / NUCDF Neonatal Onset / Executive / Program Manager / Manager Phone / site coordinator / CAO / /

Product

Buphenyl / Ravicti / /

ProgrammingLanguage

DC / /

ProvinceOrState

Minnesota / California / Colorado / Ontario / /

PublishedMedium

Baseline / /

Technology

magnetic resonance imaging / spectroscopy / MRI technology / transplantation / MRI / http / /

URL

www.CureUCD.org / www.RareDiseasesNetwork.org/ucdc / www.surveymonkey.com/s / /

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