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The strength of correlation between maximum CMAP amplitude and our primary outcome measure, the MHFMS, suggests that in non-ambulatory patients with SMA the CMAP may be a robust surrogate outcome measure for assessing treatment interventions if the putative mechanism of effect is to enhance innervated muscle mass by reinnervation or other trophic effects on nerve or muscle

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The compound remains detectable in urine for up to 4 days by mass spectrometry methods, despite its short plasma half-life, suggesting tissue distribution and binding may extend its biological effects beyond plasma clearance times

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