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A risdiplam analogue binds to the ESE2 region, which alters the stem-loop structure, TSL1, in the first half of SMN2 exon 7
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Nature Reviews Drug Discovery 2 , 114, (2003)
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Under these criteria, subjects who agreed to levocarnitine supplementation with VPA treatment were enrolled consecutively and studied prospectively from September 2013 to June 2014 at Kusatsu Hospital in Hiroshima, Japan
