A brief primer on the muscle preservation and anti-inflammatory treatment approach to Duchenne, and updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.
A brief primer on the RNA-targetd therapeutic approach to treating Duchenne, and updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.
Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, Director for Translational Research for the Advanced Heart Failure and Transplant Cardiology Program, UT Southwestern Medical Center
Associate Professor of Pediatrics / Department of Pediatric Newborn Medicine Harvard Medical School Department of Pediatrics / Brigham and Women's Hospital
Justin & Patrick are back! From wheelchair races in the courtyard to personally connecting with families, parents, caregivers and individuals with Duchenne, their experience with the community last year was hugely impactful on them. The duo is back to share new stories from their last accessible journey through Camino de Santiago and encourage others the find and foster purpose – no matter the circumstances.
Action is Therapy: Getting Involved With CureDuchenne Why I…fundraise, advocate, attend events, volunteer, engage with CureDuchenne. An overview of CureDuchenne Programs and the champions who are working to Cure Duchenne.
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?
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
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