Subcutaneous administration of TC007 reduces disease severity in an animal model of SMA.

TC007 treated mice (n = 16) lived approximately 16% longer (from an average of 15.75 ± 1.315 to 18.25 ± 0.7555 days) than vehicle treated mice (n = 16), although this difference did not reach statistical significance (Figure 1 ).

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While there was no increase in VHC cell body size, there was a slight trend towards an increased number of VHCs in treated tissues (Figure 5 ), although statistical significance was not achieved.

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