Doctors in Japan Transplant Stem Cells in Six-Day Old Newborn to Treat Urea Cycle Disorder
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Doctors in Japan Transplant Stem Cells in Six-Day Old Newborn to Treat Urea Cycle Disorder

  In an article in Medical Xpress, Japanese doctors have performed a “first” by transplanting liver cells (made with embryonic stem cells) into a newborn. The six-day-old infant was diagnosed with…

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An Ornithine Transcarbamylase Deficiency Treatment Has Been Approved for Clinical Trials
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An Ornithine Transcarbamylase Deficiency Treatment Has Been Approved for Clinical Trials

Ornithine transcarbamylase (OTC) deficiency is a disorder that can cause comas, seizures, and death, making it necessary for those affected to seek treatment. Arcturus Therapeutics aims to give those with…

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Results from Three Clinical Trials for Urea Cycle Disorders and Cirrhosis Coming by the End of the Year
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Results from Three Clinical Trials for Urea Cycle Disorders and Cirrhosis Coming by the End of the Year

Three non-investigational New Drug clinical studies are underway for Urea Cycle Disorders (UCD) and various forms of cirrhosis, initiated by Kaleido Biosciences. Dosing has been initiated in each of these…

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Man With Urea Cycle Disorder is the First Person in Canada to be Treated With Intravenous Gene Therapy
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Man With Urea Cycle Disorder is the First Person in Canada to be Treated With Intravenous Gene Therapy

According to a story from ctvnews.ca, a recent treatment with gene therapy appears to have changed the life of thirty year old Josh McQuillin, who has a urea cycle disorder,…

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CureVac Partners with Arcturus Therapeutics to Target Ornithine Transcarbamylase Deficiency
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CureVac Partners with Arcturus Therapeutics to Target Ornithine Transcarbamylase Deficiency

Another successful partnership in the rare disease community has come to fruition, reports The Pharma Letter. Just this past week, CureVac and Arcturus Therapeutics will become a powerhouse together with…

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Urea Cycle Disorders: How Deadly Bacteria Could Save Your Life
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Urea Cycle Disorders: How Deadly Bacteria Could Save Your Life

G-M-O. Three letters guaranteed to spark lively, if not heated, debate amongst lifelong friends. These three letters, of course, stand for "genetically modified organisms." Why are they so controversial? Why…

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Screen Your Baby for Urea Cycle Disorders, Get a Jump Start on Good Health
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Screen Your Baby for Urea Cycle Disorders, Get a Jump Start on Good Health

A urea cycle disorder occurs when the body can't break down the ammonia that amino acids produce. Unchecked, ammonia is toxic. According to the National Urea Cycle Disorders Foundation, there…

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