Muscular Dystrophy Association Clinical and Scientific Conference (MDA)
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.
Eteplirsen Treatment Significantly Slows Heart Function Decline in Duchenne Muscular Dystrophy
March 17th 2025A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.
Steps in Translating Genetic Discoveries Into Effective Treatments for ALS: Matthew B. Harms, MD
February 24th 2025The associate professor of neurology at Columbia University provided commentary on the rapid advancements in translating genetic research into ALS treatments and the challenges that remain. [WATCH TIME: 5 minutes]
Navigating the Future of Gene Therapy in Neuromuscular Disorders: John Brandsema, MD
February 20th 2025The pediatric neurologist at Children’s Hospital of Philadelphia provided background on a notable track session at the upcoming MDA Clinical & Scientific Conference covering updates in gene therapy for neuromuscular conditions. [WATCH TIME: 4 minutes]
Advancing Therapeutic Knowledge of Genetic Influence in ALS: Matthew B. Harms, MD
February 19th 2025The associate professor of neurology at Columbia University gave clinical insight on what neurologists and other neuromuscular specialists can expect from the ALS/frontotemporal dementia track at this year’s MDA Clinical & Scientific Conference. [WATCH TIME: 3 minutes]
ALS, Genetics, and New Horizons: What to Expect at the 2025 MDA Conference
February 18th 2025Matthew B. Harms, MD, an associate professor of neurology at Columbia University Irving Medical Center, an MDA Care Center, gave clinical insights on the advances in ALS genetic research and what clinicians can expect from the ALS/FTD track at the 2025 MDA Clinical & Scientific Conference.
Expanding the Development of Cell Therapy for Muscular Dystrophy: Rita Perlingeiro, PhD
February 18th 2025The Lillehei professor in stem cell and regenerative cardiovascular medicine at the University of Minnesota talked about the potential of investigating inducted pluripotent stem cells in neuromuscular diseases at MDA’s 2025 conference. [WATCH TIME: 8 minutes]
A Melting Pot of Strategies and Collaboration at the 2025 MDA Conference: Sharon Hesterlee, PhD
September 26th 2024The chief research officer of the Muscular Dystrophy Association talked about what is to be expected at the 2025 MDA Conference, held March 16-19, in Dallas, Texas. [WATCH TIME: 5 minutes]
Evaluating Givinostat's Efficacy and Safety in Duchenne Muscular Dystrophy: Marcello Paglione, PhD
April 3rd 2024The executive medical lead at Italfarmaco SpA talked about data from both animal models and clinical trials supporting the efficacity of givinostat in patients with Duchenne muscular dystrophy at MDA 2024. [WATCH TIME: 5 minutes]
Integrating Genetics and Transitional Support in Muscular Dystrophy Care: Amanda Peltier, MD
March 27th 2024The professor of neurology at Vanderbilt University Medical Center discussed advocating for reinstating personalized resources, expanding genetic testing, and improving transitional care in muscular dystrophy. [WATCH TIME: 5 minutes]
Promising Preclinical Evidence of Skeletal Muscle Regenerator SAT-3247: Frank Gleeson
March 26th 2024The president and chief executive officer of Satellos Bioscience provided an overview of the previously observed preclinical data supporting SAT-3247 as a potential therapy for muscular dystrophies. [WATCH TIME: 4 minutes]
A Patient Perspective on Gene Therapy for Neuromuscular Diseases: Justin Moy
March 26th 2024The second-year PhD student in bioinformatics at Boston University who lives with LAMA2 congenital muscular dystrophy talked about the potential impact and challenges of gene therapy in neuromuscular diseases. [WATCH TIME: 5 minutes]
Enhancing Multidisciplinary Care for Neuromuscular Disorders: Amanda Peltier, MD
March 25th 2024The professor of neurology at Vanderbilt University Medical Center talked about the importance of multidisciplinary care centers to improve accessibility and quality of care for patients with neuromuscular disorders. [WATCH TIME: 5 minutes]
Overviewing Phase 2 SYNAPSE-SMA Trial of NMD670 for Spinal Muscular Atrophy: Jorge Quiroz, MD, MBA
March 22nd 2024The chief medical officer of NMD Pharma provided perspective on a newly launched phase 2 study assessing NMD670, a first-in-class neuromuscular transmission enhancer, in patients with spinal muscular atrophy type 3. [WATCH TIME: 5 minutes]
Flaws in Assessment Scales for Neuromuscular Conditions: Laurent Servais, MD, PhD
March 20th 2024The professor of pediatric neuromuscular diseases at the University of Oxford provided commentary on the commonly used scales to assess neuromuscular function and where inconsistencies have been observed. [WATCH TIME: 7 minutes]
