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.
Advances in Gene Therapy for Neuromuscular Diseases: Kevin Flanigan, MDMarch 31st 2023
The 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]
The Implementation of Platform Trials for ALS Research: Melanie Quintana, PhDMarch 31st 2023
The senior statistical scientist and director of consulting at Berry Consultants talked about innovative clinical trial design in ALS research at the 2023 MDA conference. [WATCH TIME: 5 minutes]
Importance of Coordination in ALS Collaborative Research: Kuldip Dave, PhDMarch 29th 2023
The senior vice president of research at The ALS Association talked about the complexities of ALS and the importance of collaboration in research at the 2023 MDA conference. [WATCH TIME: 5 minutes]
Maximizing Exercise as Movement Medicine for Neuromuscular Diseases: Donovan Lott, PT, PhD, CSCSMarch 27th 2023
The 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, MDMarch 26th 2023
The 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]
Clinical Trial Experiences Managing Adverse Events in Gene Therapy for DMD: Natalie L. Goedeker, CPNPMarch 24th 2023
At the 2023 MDA Conference, a neurology nurse practitioner in the Neuromuscular Division at Washington University in St. Louis talked about adverse event management of gene therapies in Duchenne muscular dystrophy. [WATCH TIME: 2 minutes]
New MDA Program for Gene Therapy Development in Ultra-Rare Diseases: Sharon Hesterlee, PhDMarch 24th 2023
At 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]
Losmapimod Demonstrates Significant Slowing of Facioscapulohumeral Muscular Dystrophy Over Long-Term PeriodMarch 23rd 2023
Durability of treatment response on reachable workspace was observed among those who continued losmapimod throughout the 96-week period and those who switched from placebo.
Improving Quality of Life, Digestive Health in Duchenne Muscular Dystrophy: David Brumbaugh, MDMarch 23rd 2023
The 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 ExtensionMarch 23rd 2023
In 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, MPHMarch 23rd 2023
The 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 DMDMarch 23rd 2023
The 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.
NeuroVoices: Emma Ciafaloni, MD, on Mounting Progress in the Neuromuscular Field, Duchenne Muscular DystrophyMarch 22nd 2023
The professor of neurology and pediatrics at University of Rochester Medical Center shared her thoughts on the 2023 MDA Clinical and Scientific Conference, and the progress made in the treatment of neuromuscular disorders.
Raised Awareness and Need For Improvements to End of Life Care in Neuromuscular Disorders: Ambereen Mehta, MD, MPHMarch 21st 2023
The palliative care physician and assistant professor of medicine at Johns Hopkins Bayview Medical Center provided perspective on current state of end-of-life care for neuromuscular disorders and areas to improve upon. [WATCH TIME: 4 minutes]
The Use of Molecular Testing in Limb Girdle Muscular Dystrophy: Douglas Sproule, MD, MScMarch 21st 2023
The chief medical officer at ML Bio Solutions spoke about the advances in neuromuscular diseases such as limb girdle muscular dystrophy at the 2023 MDA Conference. [WATCH TIME: 3 minutes]
Turning Gene Therapy Into Reality: Peter Marks, MD, PhDMarch 20th 2023
The director of the Center for Biologics Evaluation Research at the FDA provided an overview of the Keynote Address he gave at the 2023 Muscular Dystrophy Association Clinical and Scientific Conference. [WATCH TIME: 3 minutes]
Minor Differences in Inclusion Criteria, Baseline Characteristics Impact Motor and Nonmotor Clinical Outcomes in Presymptomatic Spinal Muscular AtrophyMarch 20th 2023
Differences in baseline characteristics and eligible criteria in presymptomatic infants with spinal muscular atrophy led to differential results on motor and nonmotor clinical outcomes.
ALS Advances and Hot Topics for MDA 2023: Matthew B. Harms, MDMarch 9th 2023
The 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 MelmeyerJanuary 25th 2023
Ahead 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 2023January 10th 2023
Angela 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, PhDDecember 1st 2022
The 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]