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Competing Interests: Declaration of Competing Interests Lindsay N Alfano reports a relationship with Sarepta Therapeutics that includes: funding grants, speaking and lecture fees, and travel reimbursement; Astellas Gene Therapies that includes: funding grants; ATOM International, Ltd that includes: consulting or advisory fees, and travel reimbursement. Meredith K James reports a relationship with Sarepta that includes: consulting or advisory. Meredith James reports a relationship with Pfizer that includes: consulting or advisory. Meredith James reports a relationship with ATOM Ltd that includes: consulting or advisory and travel reimbursement. Gita M Ramdharry reports a relationship with Orphazyme ApS that includes: consulting or advisory. Gita Ramdharry reports a relationship with F Hoffmann-La Roche Ltd that includes: consulting or advisory. Linda P Lowes reports a relationship with Sarepta Therapeutics Inc that includes: funding grants and travel reimbursement. Linda Lowes reports a relationship with ATOM International that includes: consulting or advisory and travel reimbursement. Declarations of interests of members of the Workshop Study Group can be found online.