Save Our Sons Duchenne Foundation provided funding to Solid Biosciences to enable the development of reliable measurements of dystrophin expression, and overall muscle improvement to determine the potential clinical benefit of SGT-001.

Pre-clinical assessments of microdystrophin expression and resultant improvements in markers of muscle disease, were key components in building confidence in the potential of SGT-001 to have a meaningful benefit in Duchenne.

By collaborating with Dr. Lawlor at the Medical College of Wisconsin, Solid was able to accelerate the completion of IND-enabling studies that identified efficacious dose levels of SGT-001.  This work will serve as a foundation for analysis and interpretation of data generated from the Solid’s ongoing IGNITE-DMD clinical trial for the evaluation of SGT-001 .

Thanks to the generosity of our supporters we were able to provide this vital funding to Solid Biosciences, to enable them to continue their work to bring meaningful therapies to all patients with Duchenne.  Being able to support research projects such as these, is key to Save Our Sons Duchenne Foundation achieving our mission to find a cure for Duchenne.  Read more in the final report.

Klair Bayley – 22nd March 2019

SaveOurSons_Final_Report_SGT-001 – Microdystrophin Gene Therapy Preclinical Histological Analysis