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Disease burden quantification and outcome measures of X-linked myotubular myopathy


  • X-linked myotubular myopathy (XLMTM) is a slowly progressive disease for male survivors, however the Motor Function Measure 32 (MFM32) is sensitive to changes over one-year period.

Why this matters

  • MFM32 is an attractive outcome measure for future clinical trials of drugs which aim to stabilize people with XLMTM.

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