This fall, PPMD is presenting a gene therapy webinar series with companies and institutions who are developing therapies for Duchenne that are commonly referred to as gene therapy, including micro-dystrophin and CRISPR/Cas9.
The second webinar in this series, taking place on Wednesday, September 6 at 2 PM ET, will feature Dr. Jerry Mendell of Nationwide Children’s Hospital who will discuss his upcoming gene therapy trial, including trial design, inclusion/exclusion criteria, and timelines.
About this trial:
PPMD recently awarded our largest grant ever to Dr. Mendell, MD, PhD; co-PI Louise Rodino-Klapac, PhD; and Nationwide Children's Hospital. This grant is supporting Drs. Mendell and Rodino-Klapac and their team's work in exploring gene therapy as a potential treatment for Duchenne. Sarepta Therapeutics has also partnered in supporting Drs. Mendell and Rodino-Klapac in this exciting work.