We are dedicated to improving the lives of those touched by life-altering rare and genetically defined diseases. This commitment not only involves developing treatments for people affected by such diseases but promoting access to these treatments.
In collaboration with the rare and genetically defined disease communities we serve, we design programs that support individuals throughout their journey and assist patients and their families in exploring and accessing available treatment options. For U.S. PK deficiency patients, we run the myAgios® Patient Support Services program, which includes clinically experienced patient support managers and disease education for all patients as well as financial assistance and insurance support for people prescribed PYRUKYND®. In addition, we have a Global Managed Access Program (GMAP) aimed at providing access to PYRUKYND® in regions where the drug is not yet widely available. The GMAP was recently launched in the European Union after the approval of PYRUKYND® in this region, and EU healthcare providers may submit an inquiry on behalf of their patients.