Latest Conference Coverage

Despite Limited Literature, Combination Therapy Occurring in Spinal Muscular Atrophy

Comprised of more than 400 patients with SMA, results showed that wanting to be treated with all possible DMTs was the most common reason for combination therapy.

Raising Awareness for the Lasting Effects of Traumatic Brain Injury: Shae Datta, MD

Expanding the Access and Possibilities of Care for Adults With Neuromuscular Disorders

Amanda 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

The 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]

Impact of Indole 3-Lactate Supplementation in Multiple Sclerosis Animal Models: Larissa Jank, MD

The postdoctoral researcher in the department of neurology at Johns Hopkins School of Medicine discussed findings from a study on the impact of indole 3-lactate supplementation in animal models of multiple sclerosis. [WATCH TIME: 4 minutes]

EPIDYS Trial Supports Givinostat's Efficay, SRP-9001 in EMBARK Study, Eteplirsen Shows Long-Term Safety

Neurology News Network for the week ending March 9, 2024. [WATCH TIME: 3 minutes]

Addressing Diversity Gaps in Neuromyelitis Optica Spectrum Disorder Research: Mirla Avila, MD

The director of the Comprehensive Care MS Center at Texas Tech University Health Sciences Center talked about findings from a subgroup analysis of Hispanic or Latin patients from the N-MOmentum trial presented at ACTRIMS Forum 2024. [WATCH TIME: 3 minutes]

Switching From Anti-CD20 Therapy to Ublituximab Shows Promising Results in Phase 3b ENHANCE Trial

New data from the phase 3b ENHANCE trial presented at ACTRIMS Forum 2024 demonstrated the smooth transition from intravenous anti-CD20 therapy to ublituximab in patients with multiple sclerosis.

Real-World Effectiveness and Safety of Monoclonal Antibodies in NMOSD: Amy Kunchok, MD, PhD

The staff neurologist at the Mellen Center for Multiple Sclerosis at Cleveland Clinic talked about findings from a real-world study assessing novel monoclonal antibodies in patients with NMOSD presented at ACTRIMS Forum 2024. [WATCH TIME: 4 minutes]

Intravenous Zolgensma Shows Motor Function Improvement in Heavier Pediatric Patients With SMA

Findings 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.

Comparing Cognitive Profiles of Older Patients with Multiple Sclerosis and Alzheimer Disease: Le Hua, MD

The director of the Multiple Sclerosis Program at the Cleveland Clinic’s Lou Ruvo Center for Brain Health talked about findings on a study comparing cognitive profiles in older patients with multiple sclerosis vs Alzheimer disease. [WATCH TIME: 5 minutes]

Machine Learning Model Enhances Real-World Studies for Predicting Disability Progression in Multiple Sclerosis

A machine learning model applied to real-world data in a multiple sclerosis study increased patient inclusion for future real-world studies on assessing patient outcomes and disability progression.

Respiratory Patterns Differ in Pediatric Duchenne Muscular Dystrophy

A 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.

US National Registry Analysis Reveals Gender Disparities in Pediatric-Onset Facioscapulohumeral Muscular Dystrophy

A recent analysis of data from the US National Registry presented at MDA 2024 revealed gender disparities in pediatric-onset facioscapulohumeral muscular dystrophy, with girls experiencing more severe outcomes.

Use of the ActiMyo Sensor to Assess 95th Centile of Stride Velocity: Laurent Servais, MD, PhD

The 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]

A Case Series Shows Long-Term Efficacy and Convenience of Ravulizumab in Generalized Myasthenia Gravis

A case series presented at the MDA 2024 revealed that ravulizumab infusion intervals show promise in stabilizing symptoms and reducing exacerbations in patients with generalized myasthenia gravis.

Integrating a Business Methodology to Improving Care for ALS: Brooke Eby

At 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

A 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

The 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

In 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

A 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

A 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.

Exon-Skipping Therapy Golodirsen Prolongs Ambulation in DMD, Long-Term Study Shows

Median time to loss of ambulation for golodirsen-treated patients was 1968 days vs 1092 days for external control patients.

Vatiquinone Treatment Shows Benefit Across End Points for Friedreich Ataxia in Phase 3 MOVE-FA Trial

A new analysis of phase 3 MOVE-FA trial presented at MDA 2024 revealed promising outcomes for vatiquinone, an oral 15-lipoxygenase inhibitor, in patients with Friedreich ataxia.

Redefining Relapse in Efficacy Measures for Multiple Sclerosis Treatments: Stephen Krieger, MD; Enrique Alvarez, MD, PhD

The associate professor of neurology at Icahn School of Medicine at Mount Sinai and associate professor of neurology at University of Colorado School of Medicine talked about the potential of redefining relapses in the landscape of clinical trials for multiple sclerosis. [WATCH TIME: 3 minutes]

SRP-9001 Improves Duchenne Muscular Dystrophy Disease Trajectory Despite Failing to Meet Primary End Point in Phase 3 EMBARK Trial

Over a 52-week treatment period, treatment with SRP-9001 resulted in improvements in secondary outcomes of time to rise, micro-dystrophin expression, and 10-meter walk/run.

The Intricate Relationship Between Biological Aging and Multiple Sclerosis: Yinan Zhang, MD

The 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]

Duchenne Therapy Eteplirsen Safe in Infants as Young as 6 Months, Study Shows

Patients with Duchenne muscular dystrophy between ages of 6 and 24 months old demonstrated a similar safety profile on eteplirsen than those between 24 and 48 months of age.

Interim Real-World Data From Phase 4 EVOLVE Study Demonstrate Sustained Treatment Effect of Eteplirsen

In a real-world study, eterplirsen was safe for patients with Duchenne muscular dystrophy, with sustained or improved status in function.

Tadalafil Improves Microvascular Function in Duchenne Leg Muscles, Study Suggests

Tadalafil shows potential in addressing microvascular impairment in Duchenne muscular dystrophy, with post-contractile MRI as a screening tool.