This study is a stage 3 trial of Sareptas exon 45 and exon 53 skipping drugs. Exon skipping drugs use a small piece of genetic material to skip over a the part of the dystrophin gene with a mutation. The part of the dystrophin gene with a mutation varies between patients. This is why exon skipping trials are mutation specific.
The main objective of this study is to determine the efficacy of the drugs compared to a placebo in DMD patients. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers.
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