MARKETVUE® REPORT: Gene therapies for treatment of Glycogen Storage Disease Type 1a offer hope for overburdened patients

Ultragenyx’s AAV8 vector gene therapy, DTX401, is advancing through clinical trials as potentially the first ever therapy to treat Glycogen Storage Disease Type 1a (GSD-1a). NEWTON, Mass., Sept. 28, 2023 /PRNewswire/ — GSD-1a, also known as von Gierke’s disease, is a rare inherited…