A brief overview of the strategy guiding efforts in gene therapy and gene editing, as well as critical updates from the companies in this space. Moderated discussion included.
A brief introduction on the rationale behind mechanisms of endogenous dystrophin restoration, like exon skipping and read through agent therapies, followed by critical information and updates from the companies in this space. Moderated discussion included.
A brief overview of the rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.
This conference session provides insight and updates from companies whose therapies target critical aspects of the Duchenne disease process that occur alongside reductions in dystrophin.
An interactive presentation focused on defining and building your team at school, addressing the nuances of an IEP or 504 plan for an individual with Duchenne, and a role-playing exercise on how to effectively communicate your family’s needs in a school meeting.
This session is intended for families/caregivers of teens and adults with Duchenne. It covers four pillars of the transitional Duchenne journey: Continuity of Care, GI-related Care, Medical Privacy, and School/Career/Hobbies, and is followed by a discussion/Q&A session.
Pradeep P.A. Mammen, MD, FACC, FAHA
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
This session is intended for families/caregivers of children and adolescents with Duchenne. It covers four pillars of the early Duchenne journey: Multidisciplinary Care, Steroids, Nutrition, and Involvement in Clinical Trials, and is followed by a discussion/Q&A session.
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