A session that highlights novel and pre-clinical research in Duchenne.
Senior Vice President, Research & Development Locanabio
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.
Vice President, Patient Advocacy & External Innovation
Edgewise Therapeutics
Field Medical Director, South/Southwest Santhera Pharmaceuticals
Executive Medical Director for Medical Affairs, Neurology PTC Therapeutics
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.
Director, Center for Gene Therapy Nationwide Children's Hospital
West Medical Science Liaison Team Lead, Global Medical Affairs Sarepta Therapeutics
Executive Director, Global Medical Affairs, and Global Duchenne Muscular Dystrophy Lead PTC Therapeutics
Continued updates from relevant companies in the space. These presentations will be followed by a moderated Q&A opportunity.
Senior Medical Director, Clinical Development
Avidity Biosciences
Director in Clinical Science BioMarin Pharmaceutical Inc.
A panel discussion featuring leading healthcare providers that are making a positive impact on variety of aspects of care for Duchenne.
Associate Professor of Medicine and Co-Director
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, Neurology and Neurotherapeutics
University of Texas Southwestern
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.
Inspirational Keynote Speakers
I'll Push You
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.
Duchenne Mother, Grandmother and Advocate
4 Jake's Sake | CureDuchenne
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.
