Gene Therapy for Pompe Disease Effective in Mice, Poised for Human Trials (VIDEO)

Duke University Medical Center

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Dwight Koeberl, M.D., Ph.D., describes a new gene therapy for Pompe disease Duke Health has developed and tested in mice. Researchers have FDA approval to launch a Phase 1 human trial and are working to secure funding.

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Shawn Rocco/ Duke Health

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Please credit Shawn Rocco/ Duke Health

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