Sarepta Therapeutics has completed a new drug submission to the FDA in the United States for Casimersen, their third exon-skipping medicine for individuals with Duchenne amenable to exon 45.
- Casimersen is designed for the treatment of exon 45 amenable patients, approximately eight percent of patients with Duchenne.
- Casimersen is the third exon-skipping medicine using the Company’s proprietary PMO RNA-based platform.
Interim analysis from their ESSENCE trial demonstrated a statistically significant increase in dystrophin compared to baseline and placebo.
The ESSENCE study investigating both Golodirsen (exon 53 amendable) and Casimersen (exon 45 amendable) are currently recruiting in Canada. If you are interested in learning more about these trials, click here or speak to your neuromuscular team.
To find out more information on which mutations are potentially amenable to exon skipping click here.
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Director of Research and Advocacy