Sarepta Therapeutics Inc to Present Clinical Update for SRP-5051 Conference Call Summary

Edited Brief of SRPT.OQ conference call or presentation 7-Dec-20 1:30pm GMT

...A. DMD: Rare, fatal, neuromuscular genetic disease. 1. 2. An inherited X-linked condition that almost always affect young boys. 3. Caused by absence of dystrophin protein, which acts normally as a shock absorber within muscle. 4. Disease progression follows a predictable trajectory: a. Muscle weakness becomes increasingly noticeable between ages of three and five. b. Boys are often wheelchair-bound by the time they are pre-teen and then they experience cardiac and respiratory muscle deterioration. i. Typically leads to death in their mid-to-late 20s. B. PMO Platform: Uses a phosphorodiamidate morpholino backbone chemistry. 1. 2. Precisely targets a specific sequence of a pre-messenger RNA that enables the transmission of an internally truncated yet functional dystrophin protein. 3. Co. is currently serving Duchenne community with approved products borne from PMO platform EXONDYS 51 and VYONDYS 53 which are approved for patients amenable to skipping exons 51 and 53, respectively....

Brief