Pompe Disease: Research Studies

3 Related Result(s)

  1. 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. ...

  2. 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...

  3. 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...