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Pompe Disease: Research Studies

Be an Informed Participant

Before deciding to participate in a research study, take time to learn about clinical research, how it's conducted and your rights as a research participant. Following are some helpful resources from independent sources. Always remember that a clinical research study is research, not treatment.

Know Who to Contact

  • Eligibility: For questions about a specific study and who is eligible to participate, call or email the contact person listed for that study.
  • Your Rights: For questions about your rights as a research participant, contact the UF Institutional Review Boards at 352-273-9600.
  • Feedback: For general questions or feedback about study listings, email the UF Clinical and Translational Science Institute at UFStudies@health.ufl.edu.

Other Resources

  • ResearchMatch.org: Join a national registry of volunteers willing to be contacted about research studies.

For Research Teams

5 Related Result(s)

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  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...

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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.