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 more mild presentation. SRP-5051 is next generation eteplirsen, in that it targets the same population, those amenable to exon 51 skipping, but the compound is “charged”, meaning that its cell-penetrating capacity is increased.
A Phase 2 study is active, no longer recruiting.
This program is sponsored by Sarepta Therapeutics.
|Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)|
OPEN LABEL EXTENSION
|An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051|
Sarepta Presents at the PPMD 2019 Annual Conference
Webinar: Introducing PPMO – Sarepta TherapeuticsOn May 9, 2018, Sarepta Therapeutics, Inc. joined Parent Project Muscular Dystrophy for a webinar update to discuss PPMO, Sarepta's next generation precision RNA-targeting therapeutic platform for use in Duchenne muscular dystrophy. Sarepta also introduced their planned innovative enhancements to the trial design for their actively enrolling Phase 1 clinical trial, 5051-101, for their PPMO exon 51 candidate (SRP-5051, NCT: 03375255), and outlined their plans for future development of additional exon targets.
* This webinar recording contains edits to content requested by the company.