Pompe Disease Clinical Trial Patient Recruitment

Pompe disease patient recruitment for late-onset and classic infantile ERT and next-generation therapy trials.

Who qualifies: Patients with confirmed Pompe disease (GAA mutation) including late-onset and ERT-experienced patients.

Recruitment challenges: GAA mutation confirmation and acid alpha-glucosidase enzyme activity requirements.

Our approach: Metabolic disease center networks and Pompe patient advocacy partnerships.