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Navigating Clinical Trials
This resource offers a view of Clinical Trials 101: what they are, how they run, and how to make decisions surrounding participating in a clinical trial.
Video
Navigating Duchenne treatment: AGAMREE & Catalyst Pathways
Join us for this pre-recorded presentation and Q&A discussing to learn about a new FDA-approved treatment for Duchenne muscular dystrophy. During the session, Catalyst Pharmaceuticals will provide an overview of how this novel corticosteroid works and its efficacy in Duchenne. You will also learn about the journey to comprehensive support and access through the well-established Catalyst Pathways patient support program.
Alison Anderson, MS, CGC
Medical Affairs Director
Catalyst Pharmaceuticals
Video
Newborn Screening and Genetic Testing for Duchenne Muscular Dystrophy: Introduction for Families
This resource offers important information regarding genetic testing for newly diagnosed families.
CureDuchenne
Next Generation Technology Panel
A brief overview of the rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.
Video
NS Pharma
NS Pharma Industry Presentation:
VILTEPSO®(Viltolarsen) Overview
Shannon Lienert, MD
Medical Science Liaison, Medical Affairs
Video
Palliative Care and Duchenne Muscular Dystrophy
This resource provides an introduction to palliative care’s function, benefits, and how it fits into the multidisciplinary care model for Duchenne.
Tom McNalley, MD
Associate Professor of Pediatrics
University of California San Francisco Benioff's Children's Hospitals
Video
Pathway to Success
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.
Video
Physical Therapy: Early Intervention
This resource discusses the benefits of early intervention therapy, what to expect from a PT/OT, basic activity modifications, stretching and orthoses for children.
Video
Pillars of the Early Journey
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.
Barry Byrne, MD, PhD
Professor and Associate Chair
University of Florida, Department of Pediatrics
Diana Castro, MD
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern
Kindann Fawcett, PhD
Post Doctoral Fellow
University of Arkansas for Medical Sciences
Video
