Presentations covering therapies aiming to preserve and protect muscle in Duchenne, including anti-inflammatory agents, muscle stabilizers, and other mechanisms with relevant updates from companies in the space.
Karyn Koladicz, MD
Executive Medical Director for Medical Affairs, Neurology PTC Therapeutics
A brief introduction on the rationale behind exon skipping therapies, followed by an overview of current and investigative exon skipping therapies.
Key insights were shared by Dr. Angel Angelov, Dr. Ash Dugar, Lianna Orlando, PhD, Dr. Kevin M. Flanigan, Laura Torrente, PhD, Alayna Tress, MPH, Karin Lucas, PhD, Mahasweta Girgenrath, Phd, Jessica Duis, MD, Husam Younis, PharmD, PhD.
Gene Therapy Approaches to Treatment We covered the rationale behind gene therapy approaches for Duchenne and received updates from Pfizer, Sarepta Therapeutics, REGENXBIO Inc., and Solid BioSciences.
Dr. Peter Marks, M.D., Ph.D., director of the Center for Biologics Evaluation and Research (CBER) at the FDA, was our keynote speaker. CBER oversees the regulation and approval of all cell and gene therapies, including Sarepta’s Elevidys for 4-5-year-olds with Duchenne.
Adults with Duchenne as they explain how they have embraced their FUTURE with Duchenne. They will share their perspective on how they’ve navigated school, jobs, and relationships to live their most purposeful lives in the face of adversity.
DJ Kimble, Jake Marrazzo, Ryan Russell, PhD,
Hawken Miller
Presentations on early-stage research and development companies working on approaches to address the unmet therapeutic needs in the Duchenne community.
A Duchenne emergency case study, the steps to take, and the signs to watch for. From implementing an emergency plan and following it to effective communication with the Emergency Room to get the care you need. A review of the cautions and a proactive plan of care from specialists.
A moderated panel discussion focusing on the potential challenges and complications of AAV therapy and the clinical strategies to make dosing AAV more successful.
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