Abstract
The North Star Assessment for limb-girdle-type muscular dystrophies (NSAD) was initially developed and validated for use in clinical trials in dysferlinopathy. Due to the robust psychometric properties of this Rasch-built assessment, use of this scale in other limb girdle muscular dystrophy (LGMD) subtypes has been investigated. The purpose of our current study was to understand the ability of this scale to quantify function across the broad spectrum of abilities found in LGMD cohorts prior to implementation in clinical trials in LGMD subtypes other than LGMD R2.
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