Live Webinar: Interactive Q&A for Parents & Caregivers
August 2026
THE SPEAKERS:
KELSEY SAXON: Community Engagement Coordinator, CureDuchenne
DOUG LEVINE: Physical Therapist, CureDuchenne
This webinar, hosted by CureDuchenne and Avidity Biosciences, presents the initial data from the EXPLORE 44 clinical trial. The trial evaluates the efficacy and safety of Dalzota (AOC 1044) for treating Duchenne muscular dystrophy (DMD) in patients amenable to exon 44 skipping. Key findings include a significant increase in exon skipping and dystrophin production, with a notable reduction in creatine kinase levels, indicating potential muscle health improvements. The discussion also covers the future plans for the trial and the urgency to advance treatment options for DMD patients.
Founder and Director of the Neurology and Neuromuscular Care Center
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
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
