The Muscular Dystrophy Association (MDA) Clinical and Scientific Conference highlights unprecedented research advancements and clinical achievements in neuromuscular disorders and includes patients, physicians, researchers, and more, with topics of discussion focusing on all aspects of preclinical, translational, and clinical research and care.
Durability and Next Steps in Advancing ARCUS Gene Editing Platform in DMD: Cassandra Gorsuch, PhD
April 26th 2025The chief scientific officer at Precision Biosciences provided clinical insights on the durable potential of ARCUS gene editing and the company’s next steps toward in-human trials for Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Unveiling ARCUS Gene Editing Platform and Its Potential in DMD: Cassandra Gorsuch, PhD
April 23rd 2025The chief scientific officer at Precision Biosciences provided clinical commentary on ARCUS gene editing platform and its potential application in Duchenne muscular dystrophy through a durable, AAV-delivered therapeutic strategy. [WATCH TIME: 4 minutes]
Breaking Down Oculopharyngeal Muscular Dystrophy: From Clinical Gaps to Genetic Gains
April 22nd 2025Matthew Wicklund, MD, a professor of neurology at the University of Texas Health Science Center San Antonio, offered a clinical and translational overview of OPMD, highlighting current care challenges and the early promise of dual-action gene therapy strategies.
Michael Flanagan, PhD, on Del-zota’s Breakthrough Potential for Duchenne Muscular Dystrophy
April 17th 2025The chief scientific officer at Avidity Biosciences details the mechanism, early results, and future plans for Del-zota, an investigational therapy targeting exon 44 in Duchenne muscular dystrophy.
Next-Gen Gene Editing for Muscular Dystrophy: Exploring the ARCUS Approach
April 10th 2025Cassandra Gorsuch, PhD, chief scientific officer at Precision Biosciences, discussed the company’s ARCUS gene editing platform, its innovative approach to treating Duchenne muscular dystrophy, and promising preclinical data presented at MDA 2025.
Exploring Multimodal Approaches to Neural Regeneration: Kuldip Dave, PhD
March 31st 2025The senior vice president of research at The ALS Association talked about a multidisciplinary session presented at the 2025 MDA Conference that highlighted diverse strategies for neural repair, from pharmacological targets to noninvasive brain stimulation. [WATCH TIME: 6 minutes]
Insights into OPMD and Advancements in Genetic Research: Matthew Wicklund, MD
March 31st 2025The professor of neurology at the University of Texas Health Science Center San Antonio provided clinical insights on the genetic basis of oculopharyngeal muscular dystrophy, as well as the primary challenges in diagnosing and treating the condition. [WATCH TIME: 5 minutes]
The Urgency of Funding and Equity in Neuromuscular Research and Care: Robert Califf, MD
March 27th 2025The former FDA commissioner spoke on the need for policy and funding efforts for neuromuscular disorders, the stress on the clinical care system, and the need to acknowledge healthcare inequities. [WATCH TIME: 4 minutes]
MDA Study Highlights Lack of Psychosocial Care Services for Duchenne Muscular Dystrophy
March 23rd 2025Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.
Celebrating Milestones and Tackling New Frontiers in Pompe Disease: Priya Kishnani, MD
March 21st 2025The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]
Critical Lessons From Emergence of Enzyme Replacement Therapies in Pompe Disease: Priya Kishnani, MD
March 20th 2025The professor of pediatrics and division chief of Medical Genetics at Duke University delved into the transformative impact of enzyme replacement therapy on Pompe disease, addressing its advancements, limitations, and promising innovations shaping its future. [WATCH TIME: 2 minutes]
Breaking Barriers: Donovan Decker’s Journey in Gene Therapy and Advocacy for LGMD
March 20th 2025Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, shared his powerful journey as a patient advocate and gene therapy pioneer, shedding light on the challenges and progress in LGMD.