Latest Conference Coverage


Jenn Orthmann-Murphy, MD, PhD

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

March 18th 2024

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

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

March 18th 2024

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]


Amit Bar-Or, MD, FRCPC, FAAN, FANA

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

March 17th 2024

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]


Dimitrios Karussis, MD, PhD

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

March 16th 2024

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.


Jason Freeman, MD, MBA

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

March 15th 2024

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

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

March 14th 2024

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

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

March 14th 2024

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

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

March 14th 2024

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

Despite Limited Literature, Combination Therapy Occurring in Spinal Muscular Atrophy

March 12th 2024

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.


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

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

March 11th 2024

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

Adding to the Literature of KIF5A in ALS Pathogensis: Devesh Pant, PhD

March 11th 2024

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]


Larissa Jank, MD

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

March 10th 2024

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]


Mirla Avila, MD

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

March 8th 2024

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]


Barry A. Singer, MD

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

March 7th 2024

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.


Amy Kunchok, MD, PhD, staff neurologist at the Mellen Center for Multiple Sclerosis at Cleveland Clinic,

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

March 7th 2024

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]


Sandra P. Reyna, MD, chief scientific advisor and head of global medical engagement for SMA at Novartis

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

March 7th 2024

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.


Le Hua, MD

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

March 7th 2024

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]


Carl Marci, MD, chief clinical officer and chief psychiatrist and managing director of Mental Health and Neuroscience at OM1

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

March 7th 2024

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

Respiratory Patterns Differ in Pediatric Duchenne Muscular Dystrophy

March 6th 2024

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.


Natalie Katz, MD, PhD, assistant professor of pediatrics at Duke University Medical Center

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

March 6th 2024

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

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

March 6th 2024

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]


Raam Sambandam, MD, neurologist at Synapse Neurology

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

March 6th 2024

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

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

March 6th 2024

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]


Jason Howard, MD, pediatric orthopedic surgeon at Nemours/AI duPont Hospital for Children

Preoperative Disease-Modifying Agents Reduce Postoperative Complications in SMA Scoliosis Surgery

March 5th 2024

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

Mechanism of Action of Investigational Agent NMD670 for SMA: Jorge Quiroz, MD, MBA

March 5th 2024

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]


Amy D. Harper, MD, professor of neurology of Children’s Hospital of Richmond at Virginia Commonwealth University

Viltolarsen Shows Significant Pulmonary Function Gains in Ambulant and Nonambulant DMD

March 5th 2024

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.


Christian Werner, MD, executive director of Global Medical Affairs – Global DMD and Gene Therapy Lead at PTC Therapeutics

Meta Analysis Shows Slow Decline of Muscle Function With Ataluren for Nonsense Mutation DMD

March 5th 2024

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.


Eugenio Maria Mercuri, MD, PhD, professor of pediatric neurology and head of the Pediatric Neurology and Psychiatry Unit at the Gemelli Hospital Catholic University Foundation, in Rome, Italy

Post Hoc Analysis Shows Consistent Efficacy With Givinostat for DMD

March 5th 2024

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.

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