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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Detalhes bibliográficos
Publicado no:Nat Commun
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2018
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Article
Acesso em linha: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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