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.
Early Monitoring and Treatment for Bone Health in Duchenne Muscular Dystrophy: Meilan Rutter, MD
April 6th 2023At the 2023 MDA conference, a pediatric endocrinologist from Cincinnati Children’s Hospital talked about the unmet needs in treating bone health for patients with Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]
Personalized Treatment Approaches of Myopathies Based on Antibodies: Suur Bilicier, MD
April 5th 2023The associate professor of neurology at the University of Texas Health Science Center at Houston talked about advances in the classification of inflammatory myopathies at the recently concluded 2023 MDA conference. [WATCH TIME: 4 minutes]
Top Interviews from 2023 MDA: Expert Insights on Neuromuscular Diseases
April 4th 2023A group of experts in the care of patients with neuromuscular diseases—Douglas Sproule, MD, MSc; David Brumbaugh, MD; Sharon Hesterlee, PhD; Lauren Treat, MD; Donovan Lott, PT, PhD, CSCS—shared their perspectives on hot topics of treatment and management from the 2023 MDA conference.
The Potential of Genetic Therapies in Neuromuscular Diseases: Stephan Züchner, MD, PhD
April 4th 2023At the 2023 MDA conference, the professor for human genetics and neurology at the University of Miami Miller School of Medicine talked about the role of genetics in neuromuscular diseases and potential therapies. [WATCH TIME: 5 minutes]
Lessons Learned and Future Possibilities in ALS Collaborations: Edritz Javelosa, PhD
April 4th 2023At the 2023 MDA conference, the scientific research portfolio director at Muscular Dystrophy Association detailed a session on collaborative research in ALS and ongoing efforts in the field. [WATCH TIME: 3 minutes]
Focus on Gene Therapy and Neuroinflammation in ALS Research: Stanley H. Appel, MD
April 3rd 2023The director of the Ann Kimball and John W. Johnson Center for Cellular Therapeutics at Houston Methodist Hospital spoke on about advances in ALS research and promising therapies on the horizon at the 2023 MDA conference. [WATCH TIME: 8 minutes]
Holistic Approach to Medicine and Rehabilitation in Physical Therapy: Tina Duong, PhD, PT
April 2nd 2023The director of clinical outcomes and research development in neurology at Stanford University spoke about the paradigm shift in neuromuscular therapy interventions at the 2023 MDA conference. [WATCH TIME: 5 minutes]
Advances in Gene Therapy for Neuromuscular Diseases: Kevin Flanigan, MD
March 31st 2023The director of the center for gene therapy at Nationwide Children's Hospital talked about the challenges and opportunities of gene therapies for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]
Overcoming the Challenges of Ultra Rare Diseases, Limb Girdle Muscular Dystrophy: Peter Kang, MD
March 30th 2023At the 2023 MDA conference, the professor of neurology at the University of Minnesota talked about the current understanding and challenges of Limb Girdle muscular dystrophy from a clinical perspective. [WATCH TIME: 5 minutes]
Maximizing Exercise as Movement Medicine for Neuromuscular Diseases: Donovan Lott, PT, PhD, CSCS
March 27th 2023The research associate professor in the department of physical therapy at the University of Florida talked about physical therapy inventions for neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 4 minutes]
Standardizing Clinical Practice for End of Life Care in Neuromuscular Diseases: Lauren Treat, MD
March 26th 2023The child neurologist at the University of Colorado School of Medicine talked about her presentation on end of life care for patients with neuromuscular diseases at the 2023 MDA conference. [WATCH TIME: 5 minutes]
New MDA Program for Gene Therapy Development in Ultra-Rare Diseases: Sharon Hesterlee, PhD
March 24th 2023At the 2023 MDA Conference, the executive vice president and chief research officer of MDA talked about a new program from the MDA to tackle ultra-rare neuromuscular diseases. [WATCH TIME: 5 minutes]
Improving Quality of Life, Digestive Health in Duchenne Muscular Dystrophy: David Brumbaugh, MD
March 23rd 2023The associate professor of pediatrics at the University of Colorado School of Medicine talked at the 2023 MDA Conference about how digestive health impacts patients with Duchene Muscular Dystrophy. [WATCH TIME: 4 minutes]
Myasthenia Agent Zilucoplan Continues to Show Positive Efficacy in Long-Term Extension
March 23rd 2023In participants who received placebo during the parent studies, rapid improvements were observed as early as week 1 after switching to zilucoplan, and continued through week 12 of the extension period.
Efforts to Expand Neuropalliative Care for Neuromuscular Disorders: Ambereen Mehta, MD, MPH
March 23rd 2023The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center discussed the types of resources clinicians can pull from to expand neuropalliative care for patients with neuromuscular disorders. [WATCH TIME: 3 minutes]
Consensus Considerations Developed for SRP-9001 Adverse Event Management in DMD
March 23rd 2023The 12-person Dephi panel reviewed data from 3 clinical trials of delandistrogene maxeparvovec (SRP-9001; Sarepta), and developed considerations for the management of vomiting, myocarditis, acute liver injury, and immune-mediated myositis.