Metabolic Disorders

CPS1 Deficiency Von Gierke Disease Glycogen Storage Disease Carbamoyl Phosphate Synthetase Deficiency

FDA Grants Accelerated Approval to Genglycos for Glycogen Storage Disease Type Ia

The decision allows a one‑time AAV8 gene therapy for patients eight and older subject to two years of confirmatory data plus up to ten years of long‑term monitoring.