Performance of the upper limb module for Chinese patients with Duchenne muscular dystrophy: a new useful clinical tool to monitor the disease progress and as an outcome measure for therapeutic drug trial

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The recent development of therapeutic treatment to Duchenne muscular dystrophy (DMD), which is a common childhood neuromuscular disorder has highlighted the need to identify a valid and reliable outcome measures for planned clinical trials. This disease has a progressive deterioration from both lower limbs to trunk and upper limbs involvement from teenage onwards. Thus, outcome measures should be able to cover the disease spectrum from ambulatory to non-ambulatory stage. Majority of the existing clinical assessment tools are targeted at walking and lower limb gross motor skills and are inadequate to serve the above purposes.

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