Pompe Disease: Research Studies
3 Related Result(s)
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. ...
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...
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...
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Studies listed on this site have been approved by a UF Institutional Review Board (IRB), which works to ensure the welfare and rights of research participants as required by federal regulations. Study listings are provided by the UF Clinical and Translational Science Institute in collaboration with UF research teams and the UF IRBs.