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.
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]
Expanding the Access and Possibilities of Care for Adults With Neuromuscular Disorders
March 11th 2024Amanda Peltier, MD, a professor of neurology at Vanderbilt University Medical Center, spoke on current care needs for adults with neuromuscular disorders and the realistic ways to enhance clinical care going forward.
Adding to the Literature of KIF5A in ALS Pathogensis: Devesh Pant, PhD
March 11th 2024The instructor in the department of cell biology at Emory University provided context on a presentation at MDA 2024 regarding mutations in Kif5a in familial forms of amyotrophic lateral sclerosis. [WATCH TIME: 4 minutes]
Intravenous Zolgensma Shows Motor Function Improvement in Heavier Pediatric Patients With SMA
March 7th 2024Findings from the recent phase 3b SMART trial affirm the safety and efficacy of intravenous onasemnogene abeparvovec (Zolgensma; Novartis) in spinal muscular atrophy when patient weights range from 8.5 kg to 21 kg.
Respiratory Patterns Differ in Pediatric Duchenne Muscular Dystrophy
March 6th 2024A recent study presented at MDA 2024 highlighted the evolving respiratory patterns in pediatric patients with Duchenne muscular dystrophy, offering crucial insights for effective respiratory management in this patient population.
Use of the ActiMyo Sensor to Assess 95th Centile of Stride Velocity: Laurent Servais, MD, PhD
March 6th 2024The professor of pediatric neuromuscular diseases at the University of Oxford provided insight on a study presented at MDA 2024 assessing the use of a magneto-inertial sensor in ambulant children with Duchenne muscular dystrophy. [WATCH TIME: 4 minutes]
Preoperative Disease-Modifying Agents Reduce Postoperative Complications in SMA Scoliosis Surgery
March 5th 2024A recent study presented at MDA 2024 suggests that preoperative use of disease-modifying agents for patients with spinal muscular atrophy leads to less severe postoperative complications following scoliosis surgery.
Mechanism of Action of Investigational Agent NMD670 for SMA: Jorge Quiroz, MD, MBA
March 5th 2024The chief medical officer of NMD Pharma shed light on a newly announced phase 2 study assessing NMD670, a neuromuscular transmission enhancer, for patients with spinal muscular atrophy. [WATCH TIME: 4 minutes]
Meta Analysis Shows Slow Decline of Muscle Function With Ataluren for Nonsense Mutation DMD
March 5th 2024A new meta analysis of 3 studies presented at MDA 2024 revealed that ataluren significantly slowed the decline in muscle function for patients with nonsense mutation Duchenne muscular dystrophy.