uncovered a critical link between defects in the urea cycle, a key process in detoxifying ammonia in the body, and the development of fatty liver disease. Conducted during his time at Washington ...

Zevra Therapeutics has partnered with the National Urea Cycle Disorders Foundation to run an awareness campaign designed to ...

The company’s most advanced candidate therapy, SYNB1020, targets hyperammonemia in individuals born with urea cycle disorders ... to efficiently convert ammonia, a product of protein breakdown ...

Sandra A. Banta-Wright, MN, RNC, NNP; Robert D. Steiner, MD Neonates with profound and prolonged hyperammonemia with coma due to urea cycle defect will have had a neurological insult to the brain ...

is in Phase 1 trials for the treatment of urea cycle disorders, a 'group of severe, inherited metabolic diseases' that produces an inability to metabolize ammonia, leading to neurologic ...

A $14M grant will fund research on gene-editing therapies for rare metabolic diseases at the Perelman School of Medicine at the University of Pennsylvania (Penn) and Children's Hospital of ...