Gene Therapy and Small Disease

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Pompe Disease

Pompe Disease

Evaluation of Re-administration of Recombinant Adeno-Associated Virus Acid Alpha- Glucosidase (rAAV9-DES-hGAA) in Patients with Late-Onset Pompe Disease (LOPD)

The purpose of this research study is to test the safety of administration and re-administration of the acid alpha-glucosidase (GAA) gene into muscle.

POMPE DISEASE

Diet and Exercise in Pompe Disease

The purpose of this research study is to measure the effects of diet and exercise in people with Pompe diseases using a remote activity tracker.

POMPE DISEASE

Response to Diaphragmatic Pacing in Subjects with Pompe Disease

The purpose of this research study is to evaluate the effect of diaphragm pacing on breathing function in people with Pompe disease.

POMPE DISEASE

Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease

The purpose of this research study is to learn about the relationships between strength and endurance of the respiratory muscles and the movements generated during breathing.