Duchenne Muscular Dystrophy Clinical Symposium
On April 23 and 24, 2021, UT Southwestern's Wellstone Muscular Dystrophy Center hosted the fourth annual Duchenne/Becker Muscular Dystrophy Clinical Symposium. The symposium was held virtually via Zoom, presentations were recorded and are available with English and Spanish captions.
There were three goals of the Symposium:
- Provide an overview of genome editing and its potential application for treating DMD patients.
- Provide DMD and BMD patients and their families with a comprehensive overview of the clinical approach to caring for DMD and BMD patients.
April 23, 2021
Welcome Message and Introduction - Pradeep P.A. Mammen, MD and Rhonda Bassel-Duby, PhD, UT Southwestern
Keynote Lecture: Limitations of AAV gene therapy for DMD - Lee Sweeney, PhD, Myology Institute Director, University of Florida
The Genetics behind DMD/BMD - Markey McNutt, MD, PhD, UT Southwestern
April 24, 2021
Welcome Message and Introduction - Susan T. Iannaccone, MD and Rhonda Bassel-Duby, PhD, UT Southwestern
Wellstone Fellowships - Beverly Rothermel, PhD and Jaya Trivedi, MD, UT Southwestern
Update on CRISPR-Cas9 and DMD - Eric N. Olson, PhD, UT Southwestern
Pulmonary management of DMD/BMD in the setting of the COVID-19 pandemic - Bethany Lussier, MD, UT Southwestern
Role of gene modifiers in DMD-associated cardiomyopathy - Pradeep P.A. Mammen, MD, UT Southwestern
Arrhythmias and device therapy in Dystrophin-associated cardiomyopathy - James Daniels, MD, UT Southwestern
Keynote Lecture: Modifying steroid regimens in muscular dystrophy - Elizabeth McNally, MD, PhD, Director of Center for Genetic Medicine, Northwestern University