CureDuchenne is joined by Dr. Aravindhan Veerapandiyan, a Pediatric Neurologist from Arkansas Children’s Hospital, and Sheryl Marrazzo, a mother, grandmother and advocate in the Duchenne Community, for a 1-hour webinar on corticosteroids. This webinar discusses the role of corticosteroids in the Duchenne standards of care, highlights the effects of steroids after loss of ambulation, and includes a discussion on how corticosteroids play a role in the family unit over time.
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.
FibroGen accelerates the delivery of innovative treatments that operate at the cutting edge of cancer research. Our teams have identified and are developing potential first-in-class medicines for the treatment of life-threatening conditions such as metastatic pancreatic cancer, locally advanced pancreatic cancer (LAPC), and chemotherapy-induced anemia (CIA). We are keenly focused on leveraging the body’s natural pathways to treat cancer and its related conditions.
Bassem Elmankabadi, MD, ABWS
Executive Medical Director, Clinical Development (Fibrosis/Oncology)
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