ARTICLE | Distillery Therapeutics

Endocrine / Metabolic

December 20, 2016 7:38 PM UTC

Fruit fly studies suggest the generic mTOR inhibitor rapamycin could help treat Gaucher’s disease, which is caused by GBA mutations. In a GBA-knockout Drosophila model of Gaucher’s disease, rapamycin increased locomotor function and survival compared with vehicle. Next steps could include testing rapamycin in additional models of Gaucher’s disease...