Pompe Disease: Research Studies

5 Related Result(s)

  1. 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. You will be in this...

  2. 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...

  3. 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. ...

  4. 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. This...

  5. Effects of Immunomodulation Therapy on Anti-rhGAA Immune Response in Children with Pompe Disease Receiving rhGAA Enzyme Replacement Therapy

    The purpose of this research study is to determine the effect(s) of medications that alter the immune system on anti-rhGAA immune response in Pompe patients...