A brief overview of the rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.
This resource provides an introduction to palliative care’s function, benefits, and how it fits into the multidisciplinary care model for Duchenne.
Associate Professor of Pediatrics
University of California San Francisco Benioff's Children's Hospitals
This resource discusses managing Duchenne in a school setting: IEP vs. 504 Plans, modifications and accomodations in a school setting, and communicating with educators about Duchenne.
This resource discusses the benefits of early intervention therapy, what to expect from a PT/OT, basic activity modifications, stretching and orthoses for children.
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.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern
Post Doctoral Fellow
University of Arkansas for Medical Sciences
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.
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
Director, Payer Relations
Little Hercules Foundation
Pediatric Gastroenterologist
Nationwide Children's Hospital
RGX-202: REGENXBIO’s investigational gene therapy for the treatment of Duchenne Muscular Dystrophy presented by: Johannaz (Naz) Dastgir, DO, Senior Director, Clinical Development Lead at REGENXBIO
A program update on RGX-202, REGENXBIO’s investigational gene therapy program for the treatment of Duchenne.
Contact: Duchenne@regenxbio.com or patientadvocacy@regenxbio.com
Senior Director, Clinical Development Lead
REGENXBIO
