A natural history study of X-linked myotubular myopathy

OBJECTIVE: To define the natural history of X-linked myotubular myopathy (MTM). METHODS: We performed a cross-sectional study that included an online survey (n = 35) and a prospective, 1-year longitudinal investigation using a phone survey (n = 33). RESULTS: We ascertained data from 50 male patients...

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Vydáno v:Neurology
Hlavní autoři: Amburgey, Kimberly, Tsuchiya, Etsuko, de Chastonay, Sabine, Glueck, Michael, Alverez, Rachel, Nguyen, Cam-Tu, Rutkowski, Anne, Hornyak, Joseph, Beggs, Alan H., Dowling, James J.
Médium: Artigo
Jazyk:Inglês
Vydáno: Lippincott Williams & Wilkins 2017
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5649758/
https://ncbi.nlm.nih.gov/pubmed/28842446
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000004415
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