NeurosciencebioRxivPreprint
A new drug candidate for glycogen storage disorders enhances glycogen catabolism: Lessons from Adult Polyglucosan Body Disease models
Hilla Vaknin, Kumudesh Mishra, Jeevitha D’Souza…·Miguel Weil
This study demonstrates that 144DG11 improves survival and motor function in APBD mouse models by promoting autophagic glycogen catabolism through LAMP1 interaction.
DOI 10.1101/2021.03.18.436069