Diagnosis and Management of Duchenne Muscular Dystrophy in Children
A special issue of Children (ISSN 2227-9067). This special issue belongs to the section "Pediatric Neurology & Neurodevelopmental Disorders".
Deadline for manuscript submissions: 5 March 2026 | Viewed by 20
Special Issue Editor
Interests: neuromuscular disorders focusing on Duchenne muscular dystrophy
Special Issues, Collections and Topics in MDPI journals
Special Issue Information
Dear Colleagues,
Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular X-linked disorder caused by mutations in the dystrophin gene, leading to muscle degeneration with loss of ambulation and progressive involvement of respiratory and cardiac muscles. An enrolment of the dystrophin cerebral isoforms can be associated with intellectual disability and mental disorders. To date, different approaches targeting dystrophin restoration or acting on fibrosis and inflammation have been found to give promising results, including glucocorticoid therapy, which has been reported to slow the progression of motor, respiratory, and cardiac impairment when started in the early stages; however, conclusive pharmacological treatment is not yet available. Chronic GC is associated with several side effects including weight gain, delayed puberty with short stature, increasing emotional distress and secondary osteoporosis with increased risk of spontaneous fractures.
The aim of this Special Issue is collect reviews, original articles, case reports and case series focused on the drugs available for DMD treatment, the early identification of biomarkers in relation of the management of the bone and the pubertal development and the management of other clinical aspects regarding respiratory, cardiac and emotional/psychiatric findings.
Dr. Claudia Brogna
Guest Editor
Manuscript Submission Information
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Keywords
- DMD drugs
- muscle
- bone
- emotional aspect
- mental disorders associated with DMD
- pubertal development
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