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This instrument has been thoroughly validated and designed to be used as a drug labeling tool (via FDA criteria).
About
Summary This is a disease-specific Patient-reported outcome measure for patients with congenital, childhood-onset, or juvenile myotonic dystrophy type-1. This instrument was developed and validated through the outcome measures laboratory at the University of Rochester and based on original questions developed for adult myotonic dystrophy patients (also see UR Tech Id: 6-2189 “The Myotonic Dystrophy Health Index (MDHI)” for a more complete description). Patients (ages 5 to 7) with congenital, childhood-onset or juvenile myotonic dystrophy type-1 complete this instrument to identity the level of disease burden for their child. This instrument was designed to be used as an outcome measure during clinical trials.