Edgewise Therapeutics: Focused on Muscle Disease
Developing a novel therapeutic designed to protect muscle in Becker and Duchenne muscular dystrophy
Joanne Donovan, MD, PhD
Chief Medical Officer
Wave will provide an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular dystrophy who are amenable to exon 53 skipping, as well as an overview of the potentially registrational FORWARD-53 study.
Chief Development Officer
Wave Life Sciences
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
Join us for this pre-recorded presentation and Q&A discussing to learn about a new FDA-approved treatment for Duchenne muscular dystrophy. During the session, Catalyst Pharmaceuticals will provide an overview of how this novel corticosteroid works and its efficacy in Duchenne. You will also learn about the journey to comprehensive support and access through the well-established Catalyst Pathways patient support program.
Medical Affairs Director
Catalyst Pharmaceuticals
Presentations on early-stage research and development companies working on approaches to address the unmet therapeutic needs in the Duchenne community.
A Duchenne emergency case study, the steps to take, and the signs to watch for. From implementing an emergency plan and following it to effective communication with the Emergency Room to get the care you need. A review of the cautions and a proactive plan of care from specialists.
CureDuchenne Certified Physical Therapist
CureDuchenne Physical Therapy Program
Leading Duchenne providers discuss recommendations and advancements in their specialty and answer questions from the audience.
Pediatric Neurologist
Arkansas Children's Hospital
Pediatric Gastroenterologist
Nationwide Children's Hospital
Professor and Associate Chair
University of Florida, Department of Pediatrics
A moderated panel discussion focusing on the potential challenges and complications of AAV therapy and the clinical strategies to make dosing AAV more successful.
Professor and Associate Chair
University of Florida, Department of Pediatrics
Chief, Neuromuscular and Neurogenetic Disorders of Childhood Section National Institute of Neurological Disorders and Stroke/NIH
Director, Center for Gene Therapy Nationwide Children's Hospital
Co-Founder and Chief Scientific Officer
Kate Therapeutics
Adults with Duchenne as they explain how they have embraced their FUTURE with Duchenne. They will share their perspective on how they’ve navigated school, jobs, and relationships to live their most purposeful lives in the face of adversity.
DJ Kimble, Jake Marrazzo, Ryan Russell, PhD,
Hawken Miller
