T. Slocomb
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View article: Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy
Estimation of the Quality-of-Life Impact of X-Linked Myotubular Myopathy Open
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 rate…