This study is looking at whether we can quantify the smallest change in two outcome measures that is meaningful for families, patients and clinicians. Participation in this study includes just a one-off questionnaire which takes around 30-45 minutes to complete.
There have been an increasing number of studies in DMD. Although differences between the tests performed during the studies may be found, these differences may not be important for patients and their families. The Minimal Clinical Important Difference (MCID) is an idea developed to measure the smallest change that is considered important by patients, parents and clinicians. As more treatments are developed, there is a need to determine the MCID for other tests used in DMD studies. The North Star Ambulatory Assessment (NSAA) is a physiotherapy test for ambulant boys that involves exercises such as hopping, standing on one leg, getting up from the floor and running. The Performance of Upper Limb (PUL) is a test that measures upper limb function. Although NSAA and PUL are used in DMD studies, the patients’ and their families’ views on MCID have not been included.
This study aims to determine the MCID for these two tests using a questionnaire given to patients, families and clinicians. There will be some questions on Quality of Life questionnaires. The findings will help to understand the results of DMD studies and help to create studies that focus on patients’ real needs and their point of view.
This study is designed solely as a questionnaire for patients, parents and clinicians.
For patients the inclusion criteria are:
For parents the inclusion criteria are: