= Fully recruited
= Not yet recruiting
= Enrolling by invitation
This study is looking at the connection between the behavioural aspects of DMD and a patient's DMD gene mutation. Participants will be asked to complete an online questionnaire, which will take approximately 70mins and can be completed at multiple sittings. This is open to male DMD patients between 5 and 17yrs old.
The objective of this study is to collect data from a large cohort of individuals with DMD and BMD focusing on the neurobehavioural aspects of these conditions and their correlation to the location of the DMD gene mutation.
Depending on the timeline of Covid-19 restrictions being lifted, the next step of this study is to invite a subgroup of these participants to attend a clinic appointment where further cognitive assessments will be performed.
The study is funded by the European Commission and will involve 6 countries (Denmark, The Netherlands, France, Spain, Italy and the UK).
Recruitment in the UK will take place in two sites; UCL Great Ormond Street Institute of Child Health and Newcastle University. The UCL site will be recruiting only children with DMD and BMD, while the Newcastle site will be recruiting both children with DMD and BMD, as well as adults with BMD. Participants will have a choice regarding which site is most suitable for them.
Monika Malinova for Newcastle University
The primary outcome measure is the DAWBA questionnaire, which will be completed online and will approximately take 70 minutes that can be completed in multiple sittings. When analysed the DAWBA provides a probability of full range of diagnosis of psychological problems as defined by DSM-5.
The secondary outcome measures include;