A multicenter, retrospective medical record review of X‐linked myotubular myopathy: The recensus study

Introduction: X‐linked myotubular myopathy (XLMTM), characterized by severe hypotonia, weakness, respiratory distress, and early mortality, is rare and natural history studies are few. Methods: RECENSUS is a multicenter chart review of male XLMTM patients characterizing disease burden and unmet medi...

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Detalles Bibliográficos
Autores principales: Beggs, Alan H., Byrne, Barry J., De Chastonay, Sabine, Haselkorn, Tmirah, Hughes, Imelda, James, Emma S., Kuntz, Nancy L., Simon, Jennifer, Swanson, Lindsay C., Yang, Michele L., Yu, Zi‐Fan, Yum, Sabrina W., Prasad, Suyash
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Clinical Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5900738/
https://www.ncbi.nlm.nih.gov/pubmed/29149770
http://dx.doi.org/10.1002/mus.26018

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5900738/
https://www.ncbi.nlm.nih.gov/pubmed/29149770
http://dx.doi.org/10.1002/mus.26018

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