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]
Integrating a Business Methodology to Improving Care for ALS: Brooke Eby
March 6th 2024At MDA 2024, Brooke Eby shared her perspective as a patient with ALS and the ways to incorporate business approaches to improving clinical care and increasing enrollment for drug trials. [WATCH TIME: 5 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]
Viltolarsen Shows Significant Pulmonary Function Gains in Ambulant and Nonambulant DMD
March 5th 2024In a recent phase 2 trial analysis of viltolarsen presented at MDA 2024, findings showed improvement in forced vital capacity compared with standard care in patients with Duchenne muscular dystrophy.
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.
Post Hoc Analysis Shows Consistent Efficacy With Givinostat for DMD
March 5th 2024A recent post hoc analysis of the phase 3 EPIDYS trial presented at MDA 2024 revealed significant positive outcomes with givinostat, a histone deacetylase inhibitor, among patients with Duchenne muscular dystrophy.
The Intricate Relationship Between Biological Aging and Multiple Sclerosis: Yinan Zhang, MD
March 4th 2024The assistant professor of neurology at The Ohio State University Wexner Medical Center talked about results from an ongoing study assessing biological aging in patients with multiple sclerosis using epigenetic clocks and p16INK4a. [WATCH TIME: 6 minutes]
Reaction to 2024 MDA Legacy Award: Jeff Chamberlain, PhD
March 3rd 2024Jeff Chamberlain, PhD, a leading professor in gene therapy focused on Duchenne muscular dystrophy, shared his reaction to being named the recipient of the 2024 MDA Legacy Award, as well as the state of the DMD field currently.
Analyzing Phase 2 CALLIPER Trial of IMU-838 for Progressive MS: Robert J. Fox, MD
March 3rd 2024The staff neurologist at the Mellen Center for Multiple Sclerosis at Cleveland Clinic talked about findings from an interim analysis of the phase 2 CALLIPER trial assessing vidofludimus calcium in progressive multiple sclerosis. [WATCH TIME: 4 minutes]