Innovations in Care

Gene Therapy for Duchenne: Translating clinical guidance into meaningful patient understanding

December 10th, 2025
1:00pm EST
Live, Virtual Webinar

Gene therapy is a major step forward in treating Duchenne muscular dystrophy (DMD), offering hope to slow disease progression with a single infusion. But it’s not simple. Deciding whether—and when—to seek gene therapy involves many factors.

Join Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA) as we review guidelines for gene therapy in DMD produced in partnership with cardiologists from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION Network) and experts from 15 leading MDA and PPMD Clinical Care Centers.

What We’ll Cover:

• Current eligibility criteria for gene therapy in Duchenne muscular dystrophy and how these may apply to their child or loved one.
• Key factors in choosing a qualified treatment center, including experience with gene therapy, multidisciplinary care, and long-term follow-up capabilities.
• How to prepare for gene therapy treatment, including medical evaluations, insurance navigation, and emotional readiness for families.
• Recognizing potential side effects of gene therapy and understand strategies for monitoring and managing them safely.
• What to expect after treatment, including follow-up care, monitoring outcomes, and ongoing support and resources for families.
• Live Q&A with the presenters

Register

Meet Our Presenters:
Barry Byrne, MD, PhD, Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center, University of Florida

Diana Bharucha-Goebel, MD, Medical Director of the Gene Therapy Center of Excellence at Nationwide Children’s, Principal Investigator in the Jerry R. Mendell Center for Gene Therapy in the Abigail Wexner Research Institute, and Associate professor of Pediatrics in The Ohio State University College of Medicine

Not available to join live on December 10^th ? Register to receive an email when the webinar is available to view on-demand.

If you have questions about this webinar or other MDA programs, please contact the MDA Resource Center at 1-833-ASK-MDA1 (275-6321) or ResourceCenter@mdausa.org.

Faculty: 

Barry Byrne, MD, PhD

Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center

University of Florida

Diana Bharucha-Goebel, MD

Nationwide Children’s, Abigail Wexner Research Institute, The Ohio State University College of Medicine