CureDuchenne Link is a centralized data hub which combines clinical data, biosamples, and patient reported data for individuals with Duchenne and Becker muscular dystrophy, as well as carriers. Information and samples provided by participants are coded and available for use by approved researchers across the world.
Esta presentación se enfoca en manejar los desafíos asociados con un diagnóstico de Duchenne.
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.
Medical Affairs Director
Catalyst Pharmaceuticals
This resource offers important information regarding genetic testing for newly diagnosed families.
A brief overview of the rationale behind next generation technology, as well as presentations from the companies in this space. Moderated discussion included.
Este panel discutirá los portadores genéticos de Duchenne, cómo comunicar el diagnóstico a otros y las experiencias con la planificación familiar.
Presidenta, Coalición Latinoamericana DMD/DMB
Coalición Latinoamericana DMD/BMD
Consejera Genética Certificada
Hospital de Niños de San Antonio
This resource discusses the benefits of early intervention therapy, what to expect from a PT/OT, basic activity modifications, stretching and orthoses for children.
Esta sesión está dirigida a familias/cuidadores de niños y adolescentes con Duchenne. Cubre cuatro pilares del viaje temprano de Duchenne: atención multidisciplinaria, esteroides, nutrición y participación en ensayos clínicos, y es seguido por una sesión de discusión/preguntas y respuestas.
Post Doctoral Fellow
University of Arkansas for Medical Sciences
Professor and Associate Chair
University of Florida, Department of Pediatrics
Pediatric Neurologist
Arkansas Children's Hospital
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern
