Pompe disease is caused by mutations in lysosomal acid α-glucosidase (GAA) and patients are being treated with recombinant human α-glucosidase (rhGAA). Here the authors present the crystal structures of rhGAA and its complexes with inhibitors and a pharmacological chaperone, which is important for drug development.
- Véronique Roig-Zamboni
- Beatrice Cobucci-Ponzano
- Gerlind Sulzenbacher
