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Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy

X-linked myotubular myopathy (XLMTM) is a rare, severe, neuromuscular disorder for which novel treatments are under investigation. This study estimated quality-of-life weights (or utilities) for children with XLMTM. The state that was rated the worst described a child unable to sit and requiring inv...

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Detalles Bibliográficos
Autores principales:	Lloyd, Andrew, Aggio, Daniel, Slocomb, Ted L., Lee, Jun, Beggs, Alan H., Bilder, Deborah A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	IOS Press 2021
Materias:	Research Report
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8673490/ https://www.ncbi.nlm.nih.gov/pubmed/34250946 http://dx.doi.org/10.3233/JND-210686

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