Duchenne muscular dystrophy (DMD) is an orphan disease associated with a substantial burden on affected patients, family caregivers and society.

The Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT) is a new patient-reported outcome scale designed to measure disease progression in DMD.

We present a cost-effectiveness model framework based on the DMDSAT simulating patients across the entire lifetime of disease.