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8. Payers have extraordinary awareness of the potential clinical and economic impact of imminent gene therapies. However, their willingness to pay is driven by clinical evidence of durability and the implementation of novel financial solutions 9. DMD deprives boys of work and career aspirations, costs millions of dollars in lost income to families, and results negative tax contributions to society 10. Current DMD patient reported outcome instruments do not adequately measure potential treatment benefits of microdystrophin. implementation of the PROMIS item banks Halpern SD, Ubel PA, Caplan AL. Solid-organ transplantation in HIV-infected patients. N Engl J Med. 2002;347:284-7. CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE 12

