Duchenne muscular dystrophy (DMD) is familiar to paediatricians as the most common childhood muscular dystrophy and leads to severe disability and early death in the late teenage years if untreated. Improvements in general care, glucocorticoid corticosteroid treatment, non-invasive ventilatory support, and cardiomyopathy and scoliosis management have significantly changed the course of DMD in treated individuals, so that survival into adulthood is now a realistic possibility for most patients. This has important implications for the medical and social sectors involved in the transition to adult medical services and the provision of suitable employment and social care. Multidisciplinary team working for optimal management of DMD-specific multisystem complications is essential, and collaboration in disease specific national clinical networks is recommended. Several curative therapeutic strategies including cell and gene therapy are being pursued but are still at an experimental stage.
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Funding: The study is funded by the Department of Health.
Competing interests: Adnan Manzur is the lead clinician for the UK NorthStar Clinical Network for Paediatric Neuromuscular Disorders, which is in part funded by Muscular Dystrophy Campaign UK. Francesco Muntoni and Maria Kinali are involved in the phase I/IIa trial using morpholino antisense oligomers in DMD.
Imperial College London is the study sponsor.