Sarepta has announced encouraging interim results with their SRP-5051 exon skipping molecule for both ambulatory (walking) and non-ambulatory (non-walking) Duchenne patients amenable to exon 51 skipping ages 4-21 years.
This new second-generation exon skipping molecule in early clinical trials shows higher tissue exposure than Eteplirsen, with a favourable safety profile. They continue to test higher doses to find the maximum tolerated dose.
For any questions about participating in a clinical trial, please contact:
Defeat Duchenne Canada
Director of Research and Advocacy