A new quality of life measure for Duchenne muscular dystrophy utilizes a 14-item patient-reported outcome measures tool for use in boys and men aged 7 years and older.
Why this matters
There is a need to improve existing patient-reported outcome measures for assessing quality of life in Duchenne muscular dystrophy. This new 14-item measure is unique in its design for use across the lifetime course of muscular dystrophy progression. In addition, it is intentionally short to minimize both individual and healthcare burden and is anticipated to be a widely used clinical tool that facilitates discussion and guides management decisions.