Brain Involvement iN Dystrophinopathies (BIND)
Assess and address crucial molecular, behavioural, neurological and neuropsychological/neuropsychiatric aspects of Duchenne and Becker Muscular Dystrophy
Principal Author: Catherine Moss
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 of dystrophin’s functions in the brain, thus working towards better treatments, care and outcomes for all those living with DMD and BMD.
Most clinical experts are aware of the occurrence of brain comorbidities in a proportion of individuals affected by DMD and BMD. These X-linked recessive disorders are the result of absent or partly functioning dystrophin protein in muscles and brain. Improved standards of care and novel therapies have greatly improved the quality and quantity of life for DMD and BMD patients over the past decade.
The goals of the BIND project are to: identify brain regions that express dystrophin in the adult and developing brain to better understand their function; identify the brain comorbidities that could be corrected with a postnatal treatment intervention; to define the spectrum of brain comorbidities in DMD and BMD individuals, and how to best assess them; and to create optimal and uniform outcome measures to assess brain comorbidities in DMD/BMD.
Our objective is to achieve a deeper understanding of involvement of the brain in Duchenne and Becker muscular dystrophy, and how this impacts the lives of patients and their families.
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