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Update on the management of Duchenne muscular dystrophy
  1. Adnan Y Manzur
  1. Dubowitz Neuromuscular Centre, United Kingdom
    1. Maria Kinali
    1. Imperial College, United Kingdom
      1. Francesco Muntoni (f.muntoni{at}ic.ac.uk)
      1. Dept of Paediatrics & Neonatal Medicine, United Kingdom

        Abstract

        Duchenne Muscular Dystrophy (DMD) is familiar to paediatricians as the most common childhood muscular dystrophy, leading to severe disability and early death in late teenage years, if untreated. Improvement in general care, glucocorticoid corticosteroid treatment, non-invasive ventilatory support, cardiomyopathy and scoliosis management have significantly changed the course of the DMD in treated individuals, so that survival into adulthood is now a realistic possibility for most DMD patients. This has important implications for the medical and social sectors to provide transition to adult medical services, suitable employment, and social care. Multidisciplinary team working for optimal management of the 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 in DMD are being pursued, but these are still at an experimental stage.

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