View Navigation Search Our Site

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

Purpose

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 receiving rhGAA enzyme replacement therapy (ERT), and to understand the immune response in Pompe children treated with ERT that do not make antibodies against ERT. In addition, the study is being conducted to determine whether treating Pompe Disease with medications that affect the immune system has any effect on the overall health or disease progression in patients with Pompe disease.

Keywords

Children, Anti-rhGAA Immune Response, Pompe Disease, rhGAA Enzyme Replacement Therapy (ERT), MYOZYME

Principal Investigator

Barry J Byrne, M.D.

Department

CHILD HEALTH RESEARCH INSTITUTE

Contact Information

bbyrne@ufl.edu

3522736563

Begin a new search for other research studies

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.

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

  • HealthStreet: Health-focused services, classes and events, and opportunities to participate in research.
  • ResearchMatch.org: Join a national registry of volunteers willing to be contacted about research studies.

For Research Teams