Ultragenyx Pharma (RARE) stock is in focus as gene therapy DTX301 met its main goal in a late-stage trial for the most common ...

Nominated KRRO-121 development candidate for the potential treatment of hyperammonemia in patients with urea cycle disorders and hepatic ...

Ammonia buildup may speed hepatocellular carcinoma in mice. Read more to see how a low-protein diet could slow tumour growth.

Arginase deficiency is a rare autosomal recessive metabolic disorder that disrupts the final step of the urea cycle, impairing the conversion of arginine into urea and ornithine. In affected ...

DUBLIN--(BUSINESS WIRE)--Horizon Therapeutics plc (Nasdaq: HZNP) in partnership with Invitae Corporation (NYSE: NVTA) today announced a new urea cycle disorder (UCD) genetic testing program designed ...

A physician scientist is making strides in understanding the molecular origins of fatty liver disease, a leading cause of liver failure in the United States. An Indiana University School of Medicine ...

What determines whether astrocytes play a beneficial or detrimental role in Alzheimer’s disease? It’s surely complicated, but a study in the June 10 online Cell Metabolism lays some of the blame on ...

Boehringer Ingelheim will be commencing a new collaboration with Thoeris GmbH with the goal of advancing a first-in-class approach for the treatment of urea cycle disorders (UCDs) independent of the ...

CAMP4 Therapeutics reports promising results for CMP-SYNGAP-01 and CMP-CPS-001 in treating genetic disorders, with favorable safety data. CAMP4 Therapeutics announced promising results from its ...

The number of elderly suffering from Alzheimer’s Disease has been rapidly rising over the past decades. For a long time, scientists believed that misfolded aggregates of amyloid-beta protein ...

INDIANAPOLIS — An Indiana University School of Medicine physician scientist is making strides in understanding the molecular origins of fatty liver disease, a leading cause of liver failure in the ...