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.
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.
ALS Advances and Hot Topics for MDA 2023: Matthew B. Harms, MD
March 9th 2023The associate professor of neurology at Columbia University and medical consultant and care center director for MDA offered his insight into the latest progress in ALS treatment and some of what will be discussed at the upcoming MDA Clinical & Scientific Conference.
MDA’s Focus on Better Access for Patients With Neuromuscular Disorders: Paul Melmeyer
January 25th 2023Ahead of the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas, the vice president of public policy and advocacy at the Muscular Dystrophy Association provided an overview of the current state of access and the need for more advocates in neuromuscular disease. [WATCH TIME: 14 minutes]
Advances in Myositis and Lingering Unmet Needs Ahead of MDA 2023
January 10th 2023Angela Lek, PhD, and Tahseen Mozaffar, MD, offered their insight into myositis and the clinical needs for the population, ahead of a focused session on it, chaired by Mozzafar at the MDA’s Clinical & Scientific Conference.
The Expectations for the 2023 MDA Conference: Donald S. Wood, PhD; Sharon Hesterlee, PhD
December 1st 2022The president and CEO, as well as the executive vice president and chief research officer, of MDA, together offered an in-depth overview of what to expect from the 2023 MDA Conference, which will be held from March 19-22, 2023, in Dallas, Texas. [WATCH TIME: 12 minutes]
Gaining a Clearer Picture of Gene Therapy in Neuromuscular Disease: Carsten G. Bönnemann, MD
March 23rd 2022The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]