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9 , e1003106 (2013)

The drug is given by IV infusion in clinic or hospital settings using dosing regimens that may include two doses separated by at least 7 days or a single larger dose, depending on weight and indication

Cellular redox homeostasis and autophagy are dysregulated in hLMNA-R482L C2C12 cells To validate the results of scRNA-Seq data we re-analyzed our bulk RNA-seq dataset GSE150365 of hLMNA-C2C12 differentiating cells 18 with a focus on the regulation of metabolism, bioenergetics, and stress defense in hLMNA-R482L/WT myoblasts

Refer to the "Introduction" section of the presentation for more information about forward-looking statements and to our reports filed with the SEC for some of the factors that might cause results to differ materially from expectations