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GMaX is taking a strong leadership role in the overall discussion of market access for gene therapy by working with many of the top minds in the field to methodically build new value frameworks as next-generation tools for decision-makers. GMaX is helping ensure Sarepta can fulfill its mission of making life-changing gene therapy accessible to as many boys as possible suffering from Duchenne muscular dystrophy (DMD). This 2019-2020 Report explains the GMaX team’s strategies, insights, rationales, and corresponding research initiatives to support the US launch of microdystrophin. Owing to the extensive expertise of the GMaX team, the results of these initiatives are designed to culminate in appropriate decisions for the reimbursement of Sarepta’s DMD microdystrophin gene therapy. Katherine Gooch, PhD, MAppEpi, Executive Director, GMaX CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE 4