The unmet need of psychopharmacological intervention for neuropsychiatric and neurodevelopmental comorbidities in Duchenne muscular dystrophy: report of 5 workshops

Brain involvement is a well-recognised feature of Duchenne muscular dystrophy (DMD), presenting with intellectual disability and/or behavioural manifestations. Cumulatively, associated neurodevelopmental comorbidities affect half of the DMD population but receive little attention in standards of care recommendations and clinical practice. Access to pharmacological interventions for emotional and behavioural symptoms is limited, despite the availability of drugs with well-established efficacy. We lack information on appropriate choice, efficacy and safety of psychopharmacological interventions in DMD. Neuromuscular clinicians and neuropsychologists from eight European countries held a series of meetings between 2024 and 2025 to review the range of behavioural and emotional comorbidities observed in their respective DMD clinics and treatment strategies implemented. Each centre provided empirical evidence of the efficacy and safety of psychopharmacological interventions based on their experience. This working group identified comorbidities for which there was full consensus on drug selection, efficacy, and apparent tolerability, as well as areas with inconsistent approaches, underscoring the need for additional evidence.