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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6243013/
https://www.ncbi.nlm.nih.gov/pubmed/30451843
http://dx.doi.org/10.1038/s41467-018-07058-4

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