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Therapy of Genetic Disorders—Novel Therapies for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an inherited, progressive muscle wasting disorder caused by mutations in the dystrophin gene. An increasing variety of approaches are moving towards clinical testing that all aim to restore dystrophin production and to enhance or preserve muscle mass. Gene therap...
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2014
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4036820/ https://ncbi.nlm.nih.gov/pubmed/24883236 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40124-014-0044-x |
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