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Nationwide Children’s Hospital Begins New First-in-Human Clinical Study for DMD Using a Novel Gene Therapy Approach


CureDuchenne is very proud that Dr. Kevin Flanigan, at Nationwide Children’s Hospital, has treated two patients with gene therapy that carry the Duplication 2 mutation.

CureDuchenne funded Dr. Flanigan’s very early work on this research in 2010, by first funding the mouse model and continuing funding through the pre-clinical phase of development.

Over the last decade, CureDuchenne has contributed over $1 million to this Duplication 2 research to establish proof of concept for this very innovative research.  We are always pleased that our early funding attracts the attention of subsequent funders.  Duplication 2 is a very rare Duchenne mutation, this project is validation of our model to create meaningful treatments for everyone affected by Duchenne.

Nationwide Children’s Hospital Begins New First-in-Human Clinical Study For DMD Using A Novel Gene Therapy Approach