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 skipping additional segments of the dystrophin code called exons, the deletion can shift from an out-of-frame deletion to an in-frame deletion. Typically, an in-frame deletion results in a smaller, but still functional, dystrophin protein. This shortened protein is expected to act in a similar way to normal dystrophin, and so relieve some symptoms of Duchenne and hopefully result in a milder presentation. NS-050/​NCNP-03 is an exon skipping therapy for those amenable to exon 50 skipping.

Status

A Phase 1/2 study is not yet recruiting.

Sponsor

This program is sponsored by NS Pharma.

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