Nonclinical Exon Skipping Studies with 2'-O-Methyl Phosphorothioate Antisense Oligonucleotides in mdx and mdx-utrn-/- Mice Inspired by Clinical Trial Results.

On a functional level, there was a nonsignificant trend suggesting an increase in the 6MWD of ∼30–40 m for patients treated with weekly doses of 6 mg/kg compared with placebo for both phase 2 studies [ 29 , 35 ].

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