Case Study: developing a complex dosing drug schedule for a DART Pompe disease study

Amics Therapeutics, based in Cranbury, NJ (USA), focuses on finding treatments for a range of devastating and rare and orphan diseases. Its lead biologics program is ATB200/AT2221, a uniquely engineered Pompe disease enzyme replacement therapy that will be administered in combination with a pharmacological chaperone and tested in multiple human …