An analysis of combined clinical trial data and what it can teach about clinical trial design. What has gone right and what should be changed in the future?
Professor and Associate Chair
University of Florida, Department of Pediatrics
CureDuchenne LINK: What we are learning from your participation in research and an overview of the current data.
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.
Executive Medical Director for Medical Affairs, Neurology PTC Therapeutics
Vice President, Patient Advocacy & External Innovation
Edgewise Therapeutics
Sr VP Medical Affairs and Drug Discovery
Catalyst
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.
Director, Center for Gene Therapy Nationwide Children's Hospital
West Medical Science Liaison Team Lead, Global Medical Affairs Sarepta Therapeutics
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.
Senior Director of Clinical Science 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
Leading Duchenne providers discuss recommendations and advancements in their specialty and answer questions from the audience.
Pediatric Neurologist
Arkansas Children's Hospital
Pediatric Gastroenterologist
Nationwide Children's Hospital
Professor and Associate Chair
University of Florida, Department of Pediatrics
Presentations on early-stage research and development companies working on approaches to address the unmet therapeutic needs in the Duchenne community.
