The Duchenne Research Fund is pleased to share an update on the Parent Project Muscular Dystrophy (PPMD) project we funded in the United States to address neurobehavioural and psychosocial issues in Duchenne.
In 2021 we awarded an £80,000 grant to PPMD to support its research focusing on key areas such as learning, behaviour, depression and anxiety in Duchenne.
The team has so far completed:
- a comprehensive literature review of the field;
- a survey of 320 parents and caregivers to establish prevalence, assessment and management of neuropsychiatric or neurobehavioural problems;
- a survey of 74 physicians aiming to understand the prevalence, assessment and management practices from the clincians’ perspective.
Analysis of the data is ongoing, but key findings include:
- Clinicians surveyed estimate that 25%-75% of Duchenne patients meet criteria for diagnosis of a neuropsychiatric or neurobehavioural condition.
- However, only 10%-15% have received a formal diagnosis and appropriate treatment.
- As many as 90% of the neuromuscular clinicians surveyed do not have access to a psychiatrist to whom they can refer, and 70% do not feel comfortable prescribing medications for neuropsychiatric, emotional or behavioural concerns.
- Around 55% of clinicians have a designated psychologist and/or neuropsychologist to whom they refer, but do not have access to them in real time.
To respond to these critical gaps, PPMD is working with clinicians to develop a comprehensive screening tool to assess behaviour, emotion, learning and social symptoms and aspects of Duchenne, with the aim of guiding referral decision-making. So far more than 40 children and young people have participated in a pilot study of the tool at Arkansas Children’s Hospital.
While the work is based in the US, we hope that findings will be of benefit to all Duchenne families. We look forward to bringing you updates in the coming months, following further data analysis of the surveys, and the continuation of the pilot study.