Dr. Francessco Muntoni, MD, Great Ormond Street Institute of Child Health in London and Dr. Carsten Bonnemann, MD, National Institute of Health, brought together the four companies with ongoing microdystrophin gene therapy programs to discuss recent Serious Adverse Events (SAEs).
The objective of the working group is to share data and better understand the cause of these SAEs and how we may be able to treat those patients with genetic variants that may be at higher risk for an SAE.
This is the first presentation from this group to a number of patient organizations. This video is being shared to help you and your family understand what happened in these instances and how the companies are working to address them.
We will continue to share updates with the community from this working group as they strive for a better understanding of what is causing the immune response and work towards developing potential solutions. PPMD continues to fund research aimed at the immune response in gene therapy through our Gene Therapy Initiative in order to address challenges and realize the possibility of safely delivering gene therapy to all patients as we fight to end Duchenne.