Neurobehavioural issues in Duchenne

Tackling learning, behaviour, depression and anxiety

Although the physical challenges of Duchenne have been investigated extensively, the neurobehavioural and psychosocial issues that can result from the diagnosis are under-explored, despite their impact on everyday life for many people living with Duchenne and their families.

We have awarded an £80,000 grant to Parent Project Muscular Dystrophy (PPMD) to support its research into neurobehavioural issues in Duchenne – focusing on key areas such as learning, behaviour, depression and anxiety.

The term “neurobehavioural” relates to the way the brain affects emotion, behaviour and learning. Neurobehavioral and cognitive diagnoses are not uncommon in those living with Duchenne, but access to timely assessment, diagnosis and treatment can be difficult to obtain and varied in experience and outcome.

PPMD’s work aims to understand the intricacies of access to neurobehavioural treatment pathways affecting people living with Duchenne. Working in partnership with Certified Duchenne Care Centres in the US, the project aims to assess new and emerging knowledge in the field, and improve detection, diagnosis and treatment of conditions.

The neurobehavioral tool, developed by Natalie Truba, PhD, of Nationwide Children’s Hospital, and Molly Colvin, PhD, of Massachusetts General Hospital, will be piloted at Arkansas Children’s Hospital under guidance of Seth Sorensen PhD and Aravindhan Veerapandiyan, MD, with a goal of rolling out use of the tool to additional sites in urban, surburban, and international settings to better understand its utility and validity across a variety of populations.

Each of these efforts will collectively inform a series of care meetings diving into practices with a goal of establishing consensus for care across learning, behaviour, depression, and anxiety in Duchenne.

The work funded by DRF is part of PPMD’s new Brain Initiative, which aims to ensure all people living with Duchenne have access to appropriate neurobehavioural care.

Click here to learn more about PPMD’s work.

Clinical research

£80,000

Funded in 2021

United States

Why is this research important?

“We recognise that while there have been expansive advancements in routine clinical care alongside development of four approved therapies for the treatment of Duchenne, access to timely diagnosis and treatment for neurobehavioural conditions in Duchenne remains challenging. By optimising routine clinical care, problems with learning, behaviour, depression and anxiety have bubbled to the surface, but how we address these issues remains inconsistent and difficult to treat. We are optimistic that this new initiative will help move the needle in how we tackle these important issues that affect everyday life for people living with Duchenne. We recognise there is much work that needs to be done in this arena, and we are excited to push forward in optimising this area of care for the Duchenne community.”
Rachel Schrader

Experienced paediatric nurse practitioner and PPMD’s Vice President of Clinical Care and Education

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