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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...

詳細記述

保存先:
書誌詳細
出版年:Nat Commun
主要な著者: 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
フォーマット: Artigo
言語:Inglês
出版事項: Nature Publishing Group UK 2018
主題:
オンライン・アクセス: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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