The unmet need of psychopharmacological intervention for neuropsychiatric and neurodevelopmental comorbidities in Duchenne muscular dystrophy: report of 5 workshops
Lay Summary
Duchenne muscular dystrophy (DMD) is a common neuromuscular condition well known for its impact on muscles, but it can also affect the brain. Around half of individuals with DMD experience challenges related to brain function, such as learning difficulties, attention problems, anxiety, mood changes, or behavioural difficulties. Although these brain related features are not progressive, they are often under-recognised and not consistently addressed in everyday care. In particular, medicines that could help manage emotional and behavioural symptoms are not widely used, and families may struggle to access the right support.
This publication brings together insights from specialists across Europe who met in a series of workshops to share their clinical experience. They discussed the empirical evidence related to the use of medications to support the mental health needs in people with DMD, and how effective and safe these treatments appear in practice. The group found that for some conditions there is general agreement on which medicines can help, while in other areas approaches vary and more research is needed.
Overall, the study highlights an important gap in clinical care: many individuals with DMD could benefit from better access to mental health and behavioural treatments, including medication where appropriate. However, only a small minority can access these effective medications. It calls for clearer guidance, more research, and greater awareness so that emotional and cognitive wellbeing are treated as an essential part of DMD care, alongside physical health, helping improve quality of life for patients and their families.