The Gregory W. Fulton Neuromuscular Center
The purpose of this study is to obtain information pertaining to the occurrence of SPK-GAA antibodies and other laboratory measures in participants with Late Onset Pompe Disease (LOPD) on an enzyme replacement therapy regimen. Additionally, this study will collect retrospective laboratory and medically-relevant historic data relating to Pompe disease from participants in hopes to provide a better understanding of LOPD features to improve the design of a future phase 1/2 interventional gene therapy trial. In most cases, participants will attend a one-time clinic visit, though the option of two separate study visits may be available.
Principal Investigator: Shafeeq Ladha, MD