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

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.