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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...
Autores principales: | , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2018
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Materias: | |
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 |