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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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Podrobná bibliografie
Vydáno v:Drug Des Devel Ther
Hlavní autoři: Lim, Kenji Rowel Q, Maruyama, Rika, Yokota, Toshifumi
Médium: Artigo
Jazyk:Inglês
Vydáno: Dove Medical Press 2017
Témata:
On-line přístup: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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