Cart
×
NS Pharma
NS Pharma Industry Presentation:
VILTEPSO®(Viltolarsen) Overview
Shannon Lienert, MD
Medical Science Liaison, Medical Affairs
Video
Edgewise Therapeutics: Focused on Muscle Disease
Developing a novel therapeutic designed to protect muscle in Becker and Duchenne muscular dystrophy
Video
Edgewise CureDuchenne Workshop Presentation
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
Video
CureDuchenne Webinar with Wave Life Sciences
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.
Anne-Marie Li-Kwai-Cheung, MChem, MTOPRA, RAPS
Chief Development Officer
Wave Life Sciences
Video
Avidity Biosciences: Presentation and discussion on initial data from EXPLORE44 Clinical Trial
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.
Diana Castro, MD
Founder and Director of the Neurology and Neuromuscular Care Center
Video
Navigating Duchenne treatment: AGAMREE & Catalyst Pathways
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.
Alison Anderson, MS, CGC
Medical Affairs Director
Catalyst Pharmaceuticals
Video
2024 FUTURES New Therapeutic Approaches to Address Unmet Needs
Presentations on early-stage research and development companies working on approaches to address the unmet therapeutic needs in the Duchenne community.
Sharif Tabebordbar, PhD
Lianna Orlando, PhD
Ye Liu, PhD
Courtney Young, PhD
2024 FUTURES Preparing for the Unexpected, Responding to Emergencies
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.
Jennifer Wallace Valdes, PT
CureDuchenne Certified Physical Therapist
CureDuchenne Physical Therapy Program
Video
2024 FUTURES Multidisciplinary Proactive Care in Duchenne
Leading Duchenne providers discuss recommendations and advancements in their specialty and answer questions from the audience.
Dr. Aravindhan Veerapandiyan
Pediatric Neurologist
Arkansas Children's Hospital
Kent Williams, MD
Pediatric Gastroenterologist
Nationwide Children's Hospital
Barry Byrne, MD, PhD
Professor and Associate Chair
University of Florida, Department of Pediatrics
Video