According to DelveInsight’s analysis, the growth of the urea cycle disorders market is expected to be mainly driven by increasing prevalence, patient awareness, advancements in newborn screening ...

The National Urea Cycle Disorders Foundation, the leading patient advocacy group for people affected by urea cycle disorders (UCDs), today announced the launch of a two-year national project to ...

Under the direction of Professor Johannes Häberle, a clinician scientist and global leader in urea cycle disorders and inherited metabolic diseases, who is uniquely positioned to lead such ...

CAMP4 Therapeutics announced promising results from its SYNGAP1 and Urea Cycle Disorders (UCDs) programs at the 28th Annual Meeting of the American Society of Gene and Cell Therapy.

Preclinical data to showcase the potential of regRNA-targeting to increase protein levels in a clinically meaningful way by upregulating gene expression Interim safety data including patient ...

CAMP4 to Present New Preclinical Data from its Urea Cycle Disorders and SYNGAP1-Related Disorders Programs at the 28th American Society of Gene and Cell Therapy Annual Meeting ...

Urea Cycle Disorder (UCD) is a genetic condition that impairs the body’s ability to remove ammonia—a toxic by-product of protein metabolism—from the bloodstream. Treatment strategies primarily focus ...

About CMP-CPS-001 CMP-CPS-001 is an antisense oligonucleotide (ASO) therapeutic candidate for the treatment of UCDs targeting carbamoyl phosphate synthetase 1 (CPS1), an enzyme that catalyzes the ...

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 United States.

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 United States. By ...

Urea cycle disorders are inherited metabolic disorders makes it hard for your body to break down proteins. Learn more about symptoms, emergency treatment, and long-term management.