Clinical Research Details

Clinical Research

A Global Prospective Observational Registry of Patients with Pompe Disease

Study Description

This is an international Registry that would like to collect and evaluate outcomes on patients with Pompe. The Sponsor of the study will look at long-term safety of treatments, effectiveness of treatments, and the quality of the Pompe patients' life. They would like to collect your de-identified medical information for 5 years. 

Inclusion/Exclusion Criteria

Patients with LOPD (late onset Pompe disease) or IOPD (infantile onset Pompe disease). 

Open Enrollment

Contact Name: Amanda Prince
Contact Phone: (904) 427-7079
Contact Email: amanda.prince@jax.ufl.edu

Investigators