Breakout Session: A Steroid Discussion, One Disease, Many Paths
With more choices available, what should families consider? A panel breakout session to help families navigate the steroid journey, make informed choices, and proactively respond to side-effects.
Diana Castro, MD
Associate Professor of Pediatrics, Neurology and Neurotherapeutics
A warm welcome from Debra, Paul and Hawken, and an informative presentation on CureDuchenne’s timeline of contributions and investments, and how these inform our future vision.
This resource presents the history of Duchenne muscular dystrophy and provides a basic understanding of the genetics, management and treatment of the diesease.
Samiya Ahmad, MD
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 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.
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.
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