Live Webinar: Interactive Q&A for Parents & Caregivers
August 2026
THE SPEAKERS:
KELSEY SAXON: Community Engagement Coordinator, CureDuchenne
DOUG LEVINE: Physical Therapist, CureDuchenne
ASK ME ANYTHING: Endocrine & Bone Health for People with DBMD Dr. David Weber, Pediatric Endocrinologist at Children’s Hospital of Philadelphia (CHOP)
Wednesday, August 5, 2026
NS Pharma Industry Presentation:
VILTEPSO®(Viltolarsen) Overview
Shannon Lienert, MD
Medical Science Liaison, Medical Affairs
Developing a novel therapeutic designed to protect muscle in Becker and Duchenne muscular dystrophy
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.
