Study Details

Antibodies to AAV8 of People with Late-Onset Pompe Disease

(IRB#: IRB_00182542)

Pompe disease occurs when people do not produce enough of a protein called acid alpha-glucosidase (GAA). GAA is needed for complete breakdown of glycogen (a stored form of blood sugar) in a part of the cell called the lysosome. The absence of GAA causes glycogen to build up in the lysosomes, which damages the cells and leads to muscle weakness. The study will see if people with Late-Onset Pompe disease have antibodies to a virus called adeno associated virus (AAV8). This virus is not known to cause any disease or illness in your body. In everyday life, people are exposed to this type of virus. The study will learn more about the number of people with the disease who have antibodies to AAV8 and the antibody level changes over time, and overall health. People in the study will share current and past medications with the study team, how they feel on a day-to-day basis, and any side effects. Medical tests will also be done during the study to track the health of participants. Being in the study requires attending 6 in-home or in person study clinic visits over 4 months. Study participation lasts about 28 months.

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