GENE THERAPY WEBINAR: OCT 15 12 PM EDT
Register now for next week's gene therapy webinar with Barry Byrne MD, PhD, Christy Pacak PhD and Todd Cade PT, PhD.
Gene therapy targeting Barth syndrome holds great promise for our community yet has potential limitations and presents significant scientific, financial, manufacturing and regulatory challenges that first must be overcome.
Join this important discussion to:
• Understand the basic concepts as well as some of the complexities of gene therapy, and how it could be applied as a potential treatment for Barth syndrome;
• Be informed on the last 24 months of progress in the field of gene therapy, particularly as a specific application for treating Barth syndrome within the Powell Center;
• Explore the unique challenges related to the application of gene therapy in an ultra-rare and multi-system disease such as Barth syndrome.
All are welcome; advance registration is required!