2021 FUTURES: Keynote Panel
An inspiring conversation about living in the moment with a rare diagnosis.
An inspiring conversation about living in the moment with a rare diagnosis.
This resource presents the history of Duchenne muscular dystrophy and provides a basic understanding of the genetics, management and treatment of the diesease.
Assistant Professor of Pediatrics, Child Neurology and Sleep Medicine | Medical Director, Comprehensive Neuromuscular Center
Baylor College of Medicine | Children's Hospital of San Antonio
This panel discussion provides a robust conversation about how clinical trials intersect with family life in the Duchenne community.
Esta presentación se centra en la atención multidisciplinaria para personas con distrofia muscular de Duchenne.
Servicios para discapacitados
Universidad de Illinois
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
Astellas Industry Presentation
Senior Medical Director and Global Medical Lead
Astellas Pharma US
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
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