Expanding therapeutic options for Pompe disease: a new small molecule inhibitor of glycogen synthase 1 (GYS1) shows preclinical promise in Pompe disease

Ann Transl Med. 2024 Dec 24;12(6):123. doi: 10.21037/atm-24-135. Epub 2024 Nov 15.
No abstract available

Keywords: Glycogen synthase inhibition; Pompe disease; glycogen storage disease (GSD); glycogen synthase 1 (GYS1); lysosomal storage disease.

Publication types

  • Editorial
  • Comment