DMD+Broader+Impact

Broader Impact:

DMD is a fatal disease that is typically diagnosed around age 5. The larger impact of the cost of care and the emotional toll on family, caregivers, and the child themselves is devastating. Exon skipping may be the difference between life and death for those that are candidates for such treatment and if said treatment proves to be effective over time and with more data. Other treatment options are being explored with gene therapy as well as exon skipping technology that targets other high mutation rate areas.

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