BIND is a large-scale study involving six European countries (Denmark, France, Italy, Spain, The Netherlands, UK), with the aim of assessing the association between dystrophin isoforms and certain behaviours in patients with Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD). The UCL Great Ormond Street Institute of Child Health and the Newcastle upon Tyne Hospitals NHS Foundation Trust, along with 19 partners from Europe and Japan, are conducting a study which will look into the brain involvement in dystrophinopathies.
They are looking for boys with DMD and BMD aged 5 – 17 years and BMD patients aged 18 – 50 years to take part. The study will include completing a set of online questionnaires about the participants’ behaviour, learning, well-being and relationships. These are estimated to take up to 70 minutes, however they can be completed in multiple sittings. 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.
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.
Project Goals
The BIND project’s ambition is to elucidate the role of dystrophin in the brain. This protein is deficient in DMD and only partly functional in BMD. The project aims to develop new outcome measures that could inform the field for future clinical trials and will promote more rigorous assessment and intervention of brain comorbidities. The ultimate goal of this project is to improve understanding and measurement of dystrophin in the brain, thus working towards better treatments, care and outcomes for all those living with DMD and BMD.
Objective goals:
- Localising the isoforms that the DMD locus produces in the brain and their function;
- Improve understanding of postnatal brain restoration of the different dystrophin isoforms using preclinical models;
- Defining the spectrum of brain comorbidities in DMD and BMD individuals, and how to best assess them;
- Creating optimal and uniform outcome measures to assess brain comorbidities in DMD and BMD.
Contact Information
If you wish to have further information about this study, or if you know a young person or family who may be interested please contact:
- UCL team by emailing either Andria Papageorgiou (andriani.papageorgiou.14@ucl.ac.uk) or Isabella Vainieri (i.vainieri@ucl.ac.uk) or
- Newcastle team by emailing Monika Malinova (monika.malinova@newcastle.ac.uk)
Further information:
This project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 847826.
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