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Eteplirsen in the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy is a fatal neuromuscular disorder affecting around one in 3,500–5,000 male births that is characterized by progressive muscular deterioration. It is inherited in an X-linked recessive fashion and is caused by loss-of-function mutations in the DMD gene coding for dystrophi...
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| Udgivet i: | Drug Des Devel Ther |
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| Main Authors: | , , |
| Format: | Artigo |
| Sprog: | Inglês |
| Udgivet: |
Dove Medical Press
2017
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| Fag: | |
| Online adgang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5338848/ https://ncbi.nlm.nih.gov/pubmed/28280301 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DDDT.S97635 |
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