Abstract
Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) are a next-generation chemistry platform in which a cell-penetrating peptide is conjugated to the phosphorodiamidate morpholino oligomer (PMO) backbone, with the goal of increasing tissue penetration, exon skipping, and dystrophin production with less frequent dosing. SRP-5051 is an investigational PPMO designed to skip DMD exon 51. Here we report results from the Part A dose-finding phase of MOMENTUM (NCT04004065), an ongoing phase 2 study of SRP-5051.
Full Text 
Sign-in/Register to access full text options
Sign-in/Register to access full text options 
Published version (
Free)
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
