Urea Cycle News and Research

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Urea cycle disorders are congenital diseases that are caused by a dysfunctional urea cycle. The genetics and biochemistry of these disorders have been extensively studied. They are the result of a deficiency of one of the six enzymes that catalyze the various biochemical reactions in the urea cycle which converts ammonia to urea for removal via urine.

Depleted metabolic enzymes contribute to tumor growth in renal cancer

Kidney cancer, one of the ten most prevalent malignancies in the world, has increased in incidence over the last decade, likely due to rising obesity rates.

10 May 2018

Genevac’s HCl-resistant EZ-2 evaporator offers reliable sample preparation for neonatal screening

Genevac reports on how its hydrochloric acid (HCl) resistant EZ-2 evaporator offers significant benefits to clinical labs tasked with analyzing plasma, blood or urine samples for neonatal disorders.

9 Feb 2018

NCI selects UTSW cancer researcher to receive Outstanding Investigator Award

The National Cancer Institute has selected UT Southwestern Medical Center cancer researcher Dr. Ralph DeBerardinis to receive its prestigious Outstanding Investigator Award.

22 Sep 2017

Research suggests novel approach to treating deadly pancreatic cancer

Pancreatic cancer is now the third leading cause of cancer mortality. Its incidence is increasing in parallel with the population increase in obesity, and its five-year survival rate still hovers at just 8 to 9 percent.

16 Aug 2017

ERYTECH partners with Queen’s University to advance its product candidate for treating arginase-1 deficiency

ERYTECH Pharma, a French clinical-stage biopharmaceutical company developing innovative therapies for rare forms of cancer and orphan diseases, today announced that it has entered into a research collaboration with Queen's University in Canada to advance the preclinical development of ERYTECH's eryminase program specifically for the treatment of arginase-1 deficiency, a rare and severe metabolic disorder related to arginine metabolism.

12 Jul 2017

Discovery of early biomarkers paves way for new insight into Huntington's disease

Early warning signs of Huntington's disease have been uncovered in a sheep carrying the human HD mutation, leading the way for new insight into this devastating illness, a new study in Scientific Reports has found.

22 Feb 2017

New gene cut-and-paste methods help correct disease-causing mutation in animal model

For the first time, researchers have treated an animal model of a genetic disorder using a viral vector to deliver genome-editing components in which the disease- causing mutation has been corrected.

3 Feb 2016

Medunik announces availability of PHEBURANE drug in Canada for chronic management of UCD

Medunik Canada, a Canadian pharmaceutical company specialized in rare diseases, is pleased to announce that PHEBURANE (a tasteless oral formulation of sodium phenylbutyrate), is now available for distribution in Canada. PHEBURANE is the first drug to be approved in Canada for the chronic management of urea cycle disorders (UCD) after receiving market authorisation on January 27th, 2015.

30 Mar 2015

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus Therapeutics Inc., developing the 'next wave' of RNA medicines, today announced that Joseph Payne, President and Chief Executive Officer, is scheduled to present a corporate overview, including the company's new messenger RNA (mRNA) therapeutics pipeline, at two upcoming healthcare conferences.

7 Jan 2015

Promethera Biosciences completes €25.33 million ($31.4 million) Series C fundraising

Promethera Biosciences, a Belgian biotechnology company developing Promethera HepaStem, a cell-based therapy for the treatment of in-born errors of metabolism and acquired liver diseases, today announces the completion of its Series C fundraising round. The company has raised €25.33 million ($31.4 million), including €20.33 million in capital.

26 Nov 2014

Newly developed agent filters out toxins from body in the event of overdose

Every minute counts in the event of an overdose. ETH professor Jean-Christophe Leroux and his team have developed an agent to filter out toxins from the body more quickly and efficiently.

17 Oct 2014

Phase 2 trial results show GPB reduces hepatic encephalopathy episodes in patients with cirrhosis

Phase 2 trial results published in the March issue of Hepatology, a journal of the American Association for the Study of Liver Diseases, suggests the potential for Glycerol Phenylbutyrate (GPB) to reduce hepatic encephalopathy episodes in patients with cirrhosis, with a safety profile similar to placebo.

27 Feb 2014

Tackling orphan diseases: an interview with Damian Marron, CEO, TxCell

The exact definition of orphan diseases varies depending on where you are in the world. In the EU it's defined as a prevalence of less than 1 in 2,000, which equates to about 250,000 patients across the EU.

4 Dec 2013

Lucane Pharma, Medunik Canada to offer new therapeutic option for patients suffering from UCD

Medunik Canada, a pharmaceutical company specialized in orphan drugs and Lucane Pharma, a French niche pharmaceutical company, announced the conclusion of an exclusive collaboration agreement.

4 Dec 2013

Promethera Biosciences completes patient enrolment for first clinical trial of HepaStem

Promethera Biosciences, a Belgian biotechnology company developing Promethera HepaStem, a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorders, announces today the completion of patient enrolment for its first clinical trial.

12 Nov 2013

Polyamine-deficient diets effective as pain relief treatment for chronic and acute pain

Before and after spinal surgery, patients on a polyamine-deficient diets experienced significant relief of both chronic and acute pain, according to a study presented at the ANESTHESIOLOGY 2013 annual meeting.

14 Oct 2013

Argininosuccinic aciduria (ASA) and gene therapy: an interview with Dr Julien Baruteau, UCL Institute for Women's Health, London

This rare genetic condition can theoretically affect patients at any age but symptoms are more often observed in childhood and even uncommonly during the first days of life.

2 Sep 2013

Urea cycle disorder patient successfully treated by Promethera(R) HepaStem in Phase I/II trial in France

Promethera Biosciences, a Belgian biotechnology company developing Promethera(R) HepaStem, a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorders, announced that it has successfully treated a patient with liver progenitor cells enrolled in its Phase I/II trial.

1 Mar 2013

Hyperion Therapeutics receives FDA approval for Ravicti to treat urea cycle disorders

The U.S. Food and Drug Administration today approved Ravicti (glycerol phenylbutyrate) for the chronic management of some urea cycle disorders (UCDs) in patients ages 2 years and older.

1 Feb 2013

Alberta Children's Hospital performs first liver cell transplant in Calgary, Canada

A three-month-old Winnipeg girl has become the first patient in Canada to receive an experimental and potentially life-saving form of therapy to improve the function of her liver.

29 Nov 2012