Latest Conference Coverage

Promising Preclinical Evidence of Skeletal Muscle Regenerator SAT-3247: Frank Gleeson

The 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

The 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

The 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]

Targeting the NLRX1 Pathway for Neuroprotection in Multiple Sclerosis: Marjan Gharagozloo, PhD

The assistant professor of neurology at Johns Hopkins Medicine talked about recent research that suggests activating the NLRX1 pathway could offer neuroprotection in multiple sclerosis. [WATCH TIME: 5 minutes]

Therapeutic Avenues for Multiple Sclerosis Through Immune Cell Communication: Jeffrey Huang, PhD

The associate professor of biology at Georgetown University talked about how immune cells can communicate with brain cells to enhance repair processes, which may offer potential treatment targets for multiple sclerosis. [WATCH TIME: 5 minutes]

Clinical Benefits to Emphasizing Patient Voices in ALS: Brooke Eby

A patient advocate within the ALS community discussed the importance of incorporating patients and their voices into clinical meetings and trial design to ultimately meet treatment goals. [WATCH TIME: 4 minutes]

Overviewing Phase 2 SYNAPSE-SMA Trial of NMD670 for Spinal Muscular Atrophy: Jorge Quiroz, MD, MBA

The 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]

Episode 111: Reviewing ACTRIMS 2024

Mind Moments®, a podcast from NeurologyLive®, brings you an interview with Daniel Ontaneda, MD, PhD. [LISTEN TIME: 32 minutes]

Data Showcases Promising Mechanism of Action of GT-02287 for Parkinson Disease

Recent data highlighted the benefit of Gain Therapeutics’ GT-02287 mechanism of action in alleviating endoplasmic reticulum stress and enhancing lysosomal enzyme activity.

Clinical Significance of Visual System Modeling for Multiple Sclerosis Research: Shiv Saidha, MBBCh

The professor of neurology at Johns Hopkins Medicine talked about modeling visual systems as an area of focus in clinical trials for multiple sclerosis as it may offer insights into neurodegeneration and neuroprotection. [WATCH TIME: 6 minutes]

Combination Therapy of CST-2032/CST-107 Shows Promise in Treating Cognitive Impairment in Phase 2a Trial

A phase 2a trial demonstrated significant cognitive improvement in patients with mild cognitive impairment or mild dementia from Alzheimer or Parkinson disease through combination adrenergic activator therapy.

Desert Mapping to Promote Health Equity in Multiple Sclerosis Care: Julie Fiol, MSCN; Andreina Barnola, MD, MPH

The associate vice president of clinical Innovation and strategy and the health equity director at National MS Society talked about leveraging data mapping to bridge healthcare gaps and promote health equity in multiple sclerosis. [WATCH TIME: 4 minutes]

Flaws in Assessment Scales for Neuromuscular Conditions: Laurent Servais, MD, PhD

The 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]

NeuroVoices: Aki Ko, on mRNA Therapeutics for Duchenne Muscular Dystrophy

The chief executive officer at Elixirgen Therapeutics provided commentary on the promise of the Bobcat mRNA, and the mechanism of action for mRNA-like therapies to treat Duchenne muscular dystrophy.

Association Between Obesity and Disease Progression in Multiple Sclerosis: Lars Alfredsson, PhD

The professor of epidemiology at Karolinska Institutet talked about findings from a comprehensive study on the correlation between obesity and accelerated progression of multiple sclerosis. [WATCH TIME: 8 minutes]

Advancing Neural Repair Through Cellular Responses and Biomarkers in MS Models: Jenn Orthmann-Murphy, MD, PhD

The assistant professor of neurology in the Perelman School of Medicine at University of Pennsylvania discussed preclinical research on neural repair in multiple sclerosis at ACTRIMS Forum 2024. [WATCH TIME: 4 minutes]

Improving MS Care Through Innovative Trial Designs: Daniel Ontaneda, MD, PhD

Following the 2024 ACTRIMS Forum, the associate professor of neurology at the Cleveland Clinic Lerner College of Medicine provided insight on the need to build creative trials for multiple sclerosis treatments. [WATCH TIME: 3 minutes]

Exploring the Potential Therapeutic Avenue of BTK Inhibitors for Progressive MS: Amit Bar-Or, MD, FRCPC, FAAN, FANA

The Melissa and Paul Anderson President’s Distinguished Professor of Neurology, Perelman School of Medicine, University of Pennsylvania, discussed the promising avenue of BTK inhibitors for addressing progressive multiple sclerosis. [WATCH TIME: 5 minutes]

Repeated Intrathecal Injection of NeuroGenesis’ Mesenchymal Stem Cell Therapy Reveals Potential Benefit in Progressive MS

A recent analysis of a phase 2 trial showed that repeated intrathecal injections of MSC therapy led to significant reductions in serum biomarkers and improvements in neurological function for progressive MS.

Consistent Real-World Efficacy of Ofatumumab Therapy for Multiple Sclerosis: Jason Freeman, MD, MBA

The medial director of Novartis talked about findings from a real-world study assessing ofatumumab treatment for multiple sclerosis presented at the 2024 ACTRIMS Forum. [WATCH TIME: 3 minutes]

Overviewing Cutting-Edge Research at 2024 ACTRIMS Forum: Daniel Ontaneda, MD, PhD

The associate professor of neurology at the Cleveland Clinic Lerner College of Medicine discussed several notable presentations and topics from the 2024 ACTRIMS Forum, highlighting advances in MS care. [WATCH TIME: 9 minutes]

Mechanism of Action of SAT-3247 in Muscular Dystrophy: Frank Gleeson

The president and chief executive officer of Satellos Bioscience provided insight on a new small molecule therapy, SAT-3247, in development for patients with muscular dystrophies. [WATCH TIME: 3 minutes]

Avidity to Begin Phase 3 HARBOR Trial in Myotonic Dystrophy Following Positive MARINA Results

Over 61.1 patient-years of treatment experience, AOC 1001 was well tolerated in patients with DM1, with nausea and headache the most commonly reported adverse events.

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]