Asistencia para Familias
Esta presentación se centra en la asistencia financiera para familias en los Estados Unidos de América.
Leslie Guzman
Directora de desarrollo de programas
Programa de Asistencia Familiar de Duchenne
Esta presentación se centra en la asistencia financiera para familias en los Estados Unidos de América.
Directora de desarrollo de programas
Programa de Asistencia Familiar de Duchenne
Advancing Exon Skipping AOCs for the Treatment of Duchenne Muscular Dystrophy
Avidity Biosciences is a biotechnology company based in La Jolla, California. At Avidity, we are driven by our mission to improve the lives of people affected by diseases with limited therapeutic options, such as Duchenne muscular dystrophy (DMD). We are doing this by realizing the broad and disruptive potential of our Antibody Oligonucleotide Conjugates (AOC™) platform. AOCs combine the specificity of monoclonal antibodies and the precision of oligonucleotides. Avidity is advancing three different exon skipping AOC drugs designed to treat the root cause of DMD in individuals who are amenable to skipping exons 44, 45 or 51. Our first program in development for the treatment of DMD is called AOC 1044 and is designed to treat individuals who are amenable to exon 44 skipping treatments. We are planning to initiate our first clinical trial with AOC 1044 by the end of 2022.
Senior Medical Director, Clinical Development
Avidity Biosciences
From FUTURES 2023
Social Worker
UT Health Austin Pediatric Neurosciences at Dell Children's
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern
Post Doctoral Fellow
University of Arkansas for Medical Sciences
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.
Vice President, Patient Advocacy & External Innovation
Edgewise Therapeutics
Vice President of Clinical Development
Capricor Therapeutics
Esta presentación trata sobre Coalición Latinoamericana DMD / BMD y cómo apoyan a las familias en diferentes países.
Presidenta, Coalición Latinoamericana DMD/DMB
Coalición Latinoamericana DMD/BMD
This resource provides tips on how to effectively communicate with doctors, teachers, family members, and more while discussing your child’s care and condition.
An interactive discussion on the unique journey of caregivers, and tools to unite and strengthen the Duchenne community in their shared experience.
Una discusión interactiva sobre el viaje único de los cuidadores y herramientas para unir y fortalecer a la comunidad de Duchenne en su experiencia compartida.
El enfoque del seminario web de hoy es ayudar a educar y conectar a las familias con el panorama de los ensayos clínicos.
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
University of Texas Southwestern