Tamoxifen prolongs survival and alleviates symptoms in mice with fatal X-linked myotubular myopathy

X-linked myotubular myopathy (XLMTM, also known as XLCNM) is a severe congenital muscular disorder due to mutations in the myotubularin gene, MTM1. It is characterized by generalized hypotonia, leading to neonatal death of most patients. No specific treatment exists. Here, we show that tamoxifen, a...

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Vydáno v:Nat Commun
Hlavní autoři: Gayi, Elinam, Neff, Laurence A., Massana Muñoz, Xènia, Ismail, Hesham M., Sierra, Marta, Mercier, Thomas, Décosterd, Laurent A., Laporte, Jocelyn, Cowling, Belinda S., Dorchies, Olivier M., Scapozza, Leonardo
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group UK 2018
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6243013/
https://ncbi.nlm.nih.gov/pubmed/30451843
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-018-07058-4
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