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.

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

Liver disease therapies: an interview with Eric Halioua, CEO of Promethera Biosciences

Liver disease is a very broad family of diseases. There are two main categories of liver disease:

27 Nov 2012

Hyperion acquires worldwide rights to Ucyclyd's Ravicti

Hyperion Therapeutics, Inc. today announced that it has acquired worldwide rights to the investigational drug Ravicti from Ucyclyd Pharma, Inc., a wholly owned subsidiary of Medicis Pharmaceutical Corporation.

23 Mar 2012

ISCO discovers novel technology to produce pure hepatocyte population

International Stem Cell Corporation announces the discovery of a novel, patent-pending technology to produce unlimited numbers of liver cells (hepatocytes) that are free of contamination with potentially dangerous undifferentiated cells.

16 Jun 2011

Cytonet receives FDA orphan drug designation for liver cell infusion to treat urea cycle disorders

International biotechnology firm Cytonet, which is investigating a liver cell infusion of donated human liver cells for the treatment of urea cycle disorders in young children, announced it has been granted orphan drug designation by the U.S. Food and Drug Administration.

18 May 2011

ISCO completes first series of hepatocytes preclinical testing

International Stem Cell Corporation announces successful completion of the first series of preclinical testing of hepatocytes derived in the lab from human parthenogenetic stem cells.

27 Apr 2011

Cytonet initiates clinical trial using liver cell infusion to treat urea cycle disorders in children

International biotechnology firm Cytonet recently launched the first ever clinical trial in the United States using an investigational liver cell infusion to treat urea cycle disorders (UCD) in children. The first patient in the Phase II trial, which will seek to enroll 20 patients in 12 medical centers, is a child with citrullinemia, which is a hereditary UCD. He received six liver cell infusions on six consecutive days at Yale School of Medicine in New Haven, Conn.

15 Apr 2011

Hyperion HPN-100 phase III pivotal study for urea cycle disorders meets primary endpoint

Hyperion Therapeutics, Inc. today announced that the Company's phase III pivotal study of glycerol phenylbutyrate (HPN-100), an investigational drug for the treatment of urea cycle disorders (UCDs), met its primary endpoint. The non-inferiority study, conducted under a Special Protocol Assessment (SPA) granted by the U.S. Food and Drug Administration (FDA) in June 2009, evaluated the ammonia control of glycerol phenylbutyrate as compared to sodium phenylbutyrate (BUPHENYL®).

3 Nov 2010

Hyperion's HPN-100 granted FDA Fast Track Designation for urea cycle disorders

Hyperion Therapeutics, Inc. announced today that its investigational compound glycerol phenylbutyrate (HPN-100) has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) as adjunctive therapy for the chronic management of urea cycle disorders. Hyperion recently completed its phase III trial which was conducted under a Special Protocol Assessment (SPA) agreement with the FDA. The Company plans to announce results from that trial later this year.

18 Oct 2010

Hyperion Therapeutics enrolls first patient in HPN-100 phase III clinical trial for urea cycle disorders

Hyperion Therapeutics today announced that the first patient has been enrolled in its pivotal phase III clinical trial of investigational compound HPN-100 (glycerol phenylbutyrate). The 4-week, multi-center, randomized, double-blind, cross-over study is designed to evaluate the non-inferiority of glycerol phenylbutyrate to BUPHENYL® (sodium phenylbutyrate) Tablets and Powder in adults with urea cycle disorders.

27 Oct 2009

Hyperion enrolls first patient in HPN-100 investigational compound clinical trial

Hyperion Therapeutics today announced that the first patient has been enrolled in a clinical trial of investigational compound HPN-100 (glycerol phenylbutyrate) in children ages 6-17 with urea cycle disorders (UCD). The study will evaluate the safety, tolerability, and ammonia control of glycerol phenylbutyrate compared to BUPHENYL® (sodium phenylbutyrate).

15 Sep 2009

Drug sodium phenylbutyrate could improve the memory of those with Alzheimer's disease

The results of the project, developed by researchers of the Center for Applied Medical Research (CIMA) of the University of Navarra have been published in the journal Neuropsychopharmacology.

20 Feb 2009

New hope for leptin as a solution to obesity

The discovery more than a decade ago of leptin, an appetite-suppressing hormone secreted by fat tissue, generated headlines and great hopes for an effective treatment for obesity.

6 Jan 2009

Watermelon provides Viagra-like effects

A cold slice of watermelon has long been a Fourth of July holiday staple. But according to recent studies, the juicy fruit may be better suited for Valentine's Day.

30 Jun 2008

Helix BioPharma awarded U.S. patent for adjunct chemotherapy applications

Helix BioPharma Corp. has announced the issuance of its second DOS47 patent (U.S. Patent # 7,264,800) from the United States Patent & Trademark Office, describing a method and composition for combining targeted DOS47 therapeutics with weakly basic chemotherapeutic drugs in adjunct treatment applications.

18 Sep 2007

FDA designates Carbaglu for treatment of NAGS deficiency as a Fast Track product

The Food and Drug Administration (FDA) in the USA has concluded that Carbaglu (carglumic acid) for treatment of N-acetylglutamate synthase (NAGS) deficiency meets the criteria for fast track designation.

10 Jul 2007

Once-fatal metabolic disorders treatable

People with a class of rare genetic disorders that often lead to brain damage, coma and death can be successfully treated with drugs, says a researcher at the Stanford University School of Medicine and Lucile Packard Children's Hospital.

1 Jun 2007

Treatment for urea cycle disorders proves its worth 25 years later

The simple notion of copying the body's own natural "waste disposal" chemistry to mop up potentially toxic nitrogen has saved an estimated 80 percent of patients with urea cycle disorders --- most of them children, according to a report in the New England Journal of Medicine summarizing a quarter century of experience with the treatment.

1 Jun 2007

Digesting protein may be a hidden problem for some

Protein metabolism presents some serious waste management challenges for the body.

27 Apr 2007

Technique for inserting genes into specific sites on the genome in liver cells

The genes are inserted into non-coding regions of the genome so there is no danger of interfering with the functioning of other genes. Once inserted, the gene remains a permanent part of the cell's genome.

10 Oct 2005