One of the most common types of mutations in the dystrophin gene occurs when a piece of the code in the middle of the gene is missing or deleted. By leaving out or skipping additional segments of the dystrophin code called exons in the muscle cell’s working copy of the gene, the ragged edges of the deletion can be pulled back together to make a smaller, but still functional, dystrophin protein. This research is designed to skip exon 45 in the dystrophin gene.
A Phase 1/2 study is completed. If positive results are achieved, global next steps will be considered.
This program is sponsored by Daiichi Sankyo in Japan.
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Daiichi-Sankyo Presents at the PPMD 2020 Virtual Annual ConferencePre-recorded content for PPMD's 2020 Virtual Annual Conference On-Demand Library