Nusinersen ameliorates motor function and prevents motoneuron Cajal body disassembly and abnormal poly(A) RNA distribution in a SMA mouse model.

We noticed that nusinersen treatment had a highly significant impact on body weight gain with a growth curve comparable to that of vehicle-treated WT (hereafter referred to as WT) mice from P6 to P12 (Fig. 1 B–D).

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