Synlogic announced the presentation of the expanded clinical data set from its first-in-human Phase 1 study of SYNB1020, a Synthetic Biotic medicine being developed for the treatment of hyperammonemia associated with urea cycle disorders
, or UCDs, and cirrhosis.
SYNB1020, is a novel, first-in-class, synthetic biotic medicine that is orally delivered and designed to treat elevated blood ammonia levels, or hyperammonemia, in genetic urea cycle disorders
(UDDs) or in chronic liver disease.
The website's focus was going to be about arginase deficiency, as well as urea cycle disorders
Belgium-based Promethera Biosciences SA reported on Monday that Health Canada has accepted its New Drug Submission (NDS) of Heparesc in neonatal onset Urea Cycle Disorders
(UCD) for review.
Urea cycle disorders
are a group of diseases associated with hyperammonemia, which causes severe neurological sequelae, seizures and psychomotor retardation.
For more information on urea cycle disorders
, visit http://www.
05 January 2017 - Cambridge, Mass-based biotechnology company RaNA Therapeutics has acquired the MRT mRNA therapy platform of Dublin, Ireland-based specialty biopharmaceutical company Shire plc (NASDAQ: SHPG) to advance programs in cystic fibrosis and urea cycle disorders
, the company said.
inhibition of the urea cycle by valproic acid), hemolytic disease (release of ammonia from red blood cells), gastrointestinal bleeds (increased ammonia generation due to microbial catabolism of hemoglobin), or urea cycle disorders
(defective or decreased urea cycle enzymes) (1).
These range from orphan indications, such as rare inborn metabolic diseases including urea cycle disorders
(UCD) to large indications such as acute-on-chronic liver failure (ACLF), and nonalcoholic steato-hepatits (NASH)/fibrosis.
Cytonet, Weinheim, Germany announced in January that the European Medicines Agency (EMA) has validated its Marketing Authorization Application (MAA) seeking approval of its liver cell therapy for the treatment of Urea Cycle Disorders
(UCD) in children.
Glycerol phenylbutyrate, a nitrogen-binding agent, has been approved for the chronic treatment of adults and children aged 2 years and older with urea cycle disorders
, who cannot be managed with a protein-restricted diet or amino acid supplements alone, according to the Food and Drug Administration.
As per the reports, Hyperion Therapeutics, a biotech focused on the treatment of urea cycle disorders
and hepatic encephalopathy, has announced terms for its IPO.