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.
Click here to read the full press release.
This trial is still recruiting at London Health Sciences Centre in London, Ontario. If you would like to learn more about this trial, click here or contact Chelsea.Huisman@lhsc.on.ca.
For any questions about participating in a clinical trial, please contact:
Defeat Duchenne Canada
Director of Research and Advocacy