PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies ...

We also expect to report dystrophin production data from the ... a favorable emerging safety profile and robust, dose-dependent splicing correction in the 5 and 10 mg/kg dose cohorts.

Elevidys was granted expanded approval by the U.S. Food and Drug Administration in 2024, with expectations high that the therapy, designed to restore a critical muscle protein called dystrophin ...

during the process of RNA splicing, causing the body to produce more dystrophin. One of the few other therapeutics focused on exon 53 skipping is NS Pharma’s Viltepso. At present, the market for ...

DMD is an X-linked disorder characterized by progressive muscle weakness and wasting due to the absence of a protein called dystrophin, which in turn causes degeneration of skeletal and cardiac muscle ...