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- W2503232171 endingPage "171" @default.
- W2503232171 startingPage "147" @default.
- W2503232171 abstract "Abstract Muscular dystrophies are a group of hereditary disorders caused by mutations in genes that have essential roles in muscle function and are generally characterized by progressive muscle weakness and degeneration. Among those, Duchenne muscular dystrophy (DMD) is the most common and severe. DMD is a fatal, X-linked recessive disorder which affects 1 in 3500 boys and is caused by mutations in the dystrophin gene. Currently there is no cure for the disorder and treatment options are limited to physiotherapy and corticosteroids that slow the course of the disorder, but are also associated with severe side effects when administered over prolonged periods of time. This chapter will provide an overview of the approaches currently being pursued for the treatment of DMD and the state of the art strategies that are in clinical trials. Emphasis will be placed on describing the progress made over the years to reach the current state, as well as the challenges that the field of gene and molecular therapies face to ensure that the approach is safe to use in patients and effective." @default.
- W2503232171 created "2016-08-23" @default.
- W2503232171 creator A5022177255 @default.
- W2503232171 creator A5082623325 @default.
- W2503232171 date "2015-01-01" @default.
- W2503232171 modified "2023-09-27" @default.
- W2503232171 title "Genetic and Cell-Mediated Therapies for Duchenne Muscular Dystrophy" @default.
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