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, Monzer Marisat, Uri Sprecher, Shane Wald-Altman, Anna Dukhovny, Yuval Raviv, Benny Da’adoosh, Hamutal Engel, Sandrine Benhamron, Keren Nitzan, Anna Permyakova, Hanna Rosenmann, Alexander Lossos, , Miguel Weil
This study demonstrates that 144DG11 improves survival and motor function in APBD mouse models by promoting autophagic glycogen catabolism through LAMP1 interaction.
bioRxiv · Mar 18, 2021 · PreprintNeuroscience