Musunuru provided ESGCT delegates with promising news of a roadmap following the diagnosis and treatment of Baby KJ.

ACER-001, a nitrogen-binding agent, is a powder formulation of sodium phenylbutyrate designed using a microencapsulation process. The Food and Drug Administration (FDA) has accepted for filing the New ...

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 that ...