Scientists achieve breakthrough in liver research with mini organoids that grow a million-fold and stay functional for months.

The liver is the body's control tower for metabolism, powering vital functions like converting nutrients to glucose, storing ...

By identifying the critical role the urea cycle plays in its development, his findings could pave the way for new medications to treat this currently incurable disease. In a study recently ...

Many diseases are caused by dysfunctional gene expression that leads to too much or too little of a given protein. Efforts to ...

Phase 1 clinical trial of CMP-CPS-001 in Urea Cycle Disorders (UCDs) ongoing, with dosing completed in two of four multiple ascending dose (MAD) cohorts; safety, pharmacokinetic, and ...

The firm focuses on the treatment of rare neurological and sleep conditions such as certain Urea Cycle Disorders, Niemann Pick Disease Type C and Idiopathic Hypersomnia. The company receives ...

Nutritional supplement for infants and toddlers with urea cycle disorder, gyrate atrophy of the choroid and retina, or hyperammonemia-hyperornithinemia-homocitrullinemia (HHH) syndrome.