Current treatment options for muscular dystrophies

The increasing understanding of the molecular mechanisms in Duchenne muscular dystrophy, which is caused by a defect in the dystrophin gene and other muscular dystrophies leads to the fact that a large number of new treatment options have found their way into clinical studies and, in some cases into clinical treatment. In addition to the strategy of replacing the missing or structurally disturbed protein, secondary phenomena such as inflammation or fibrosis are also being investigated. Despite this progress in the development of causally oriented treatment, the optimal symptomatic treatment continues to play a central role. For this purpose well-established standards of care have been published. Close cooperation between neuropediatricians, cardiologists, orthopedists and respiratory specialists is necessary in order to provide patients with adequate care.