A Holistic View of Clinical Trials
This panel discussion provides a robust conversation about how clinical trials intersect with family life in the Duchenne community.
This panel discussion provides a robust conversation about how clinical trials intersect with family life in the Duchenne community.
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
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.
This resource presents advice and resources on how to communicate a Duchenne diagnosis to your family and children.
An interactive presentation focused on harnessing creativity in day-to-day life with inclusive activities, games and exercises, as well as utilizing adaptive technology/equipment to surpass expectations.
CureDuchenne Certified Physical Therapist
CureDuchenne Physical Therapy Program
Occupational Therapist III
Children’s Health and Rehabilitation Services
Quantum Product & Technical Education Manager
Pride Mobility
Paul, Debra and Hawken Miller discuss FUTURES 2022, current advancements in therapies and next generation investments as well as programs CureDuchenne offers to improve the quality of life for those impacted by Duchenne.