Aim An international C linical O utcomes G roup consisting of clinicians, scientists, patient
advocacy groups, and industries identified a need for a scale to measure motor performance …

The recent development of therapeutic approaches for Duchenne muscular dystrophy
(DMD) has highlighted the need to identify clinical outcome measures for planned efficacy …

Background Duchenne muscular dystrophy is caused by dystrophin deficiency and muscle
deterioration and preferentially affects boys. Antisense-oligonucleotide-induced exon …

Aim Duchenne muscular dystrophy (DMD) is associated with neuropsychiatric disorders.
The aim of the study was to characterize the DMD neuropsychiatric profile fully and to …

Objective To assess the current use of glucocorticoids (GCs) in Duchenne muscular
dystrophy in the UK, and compare the benefits and the adverse events of daily versus …

OBJECTIVES: To assess attention deficit hyperactivity disorder (ADHD) in boys affected by
Duchenne muscular dystrophy (DMD) and to explore the relationship with cognitive abilities …

Artificial intelligence has the potential to revolutionize healthcare, yet clinical trials in
neurological diseases continue to rely on subjective, semiquantitative and motivation …

Aim We report four cases of acquired severe encephalopathy with massive hyperkinesia,
marked neurological and cognitive regression, sleep disturbance, prolonged mutism, and a …

Friedreichʼs ataxia (FA) is caused by a variant of the Frataxin (FXN) gene, leading to its
downregulation and progressively impaired cardiac and neurological function. Current gold …

Objective With the emergence of experimental therapies for Duchenne muscular dystrophy
(DMD), it is fundamental to understand the natural history of this disorder to properly design …