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