Hiroki, Age 26
Living with Pompe Disease
A Thank You to All Participants: At Amicus, we believe that all study participants are equal clinical research partners in the drug development process. Without the support of their families, friends, and healthcare teams, the Amicus clinical research program could not move forward. Amicus would like to express its gratitude to the Pompe community and all involved in the ongoing and completed clinical trials and all of our clinical research.
AT-GAA: Phase 1/2 Safety Study (ATB200-02 Study)
More Information:
www.clinicaltrials.gov: NCT02675465
AT-GAA: Phase 3 PROPEL Study (ATB200-03 Study)
More Information:
www.clinicaltrials.gov: NCT03729362
Amicus announced a major collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn) to pursue research and development of novel gene therapies for Pompe disease. This relationship will combine Amicus’ protein engineering and glycobiology expertise with Penn’s adeno associated virus (AAV) gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability.