Latest Conference Coverage

Exploring New Avenues for Treating Muscular Dystrophies With Targeted Therapies: Matthew Alexander, PhD

The associate professor of pediatric neurology and genetics at the University of Alabama Birmingham provided clinical insights on the key signaling pathways currently being targeted in the treatment of muscular dystrophies. [WATCH TIME: 4 minutes]

Differentiating Cognitive Decline in Multiple Sclerosis and Alzheimer Disease: Sarah Levy, PhD

The assistant professor in the department of neurology at Mount Sinai discussed distinguishing cognitive impairment in MS from AD emphasizing orientation as a key differentiator. [WATCH TIME: 5 minutes]

Duchenne Gene Therapy Delandistrogene Moxeparvovec Shows Manageable Safety Over 5-Year Period

Pooled safety data of delandistrogene moxeparvovec, a gene transfer therapy for Duchenne muscular dystrophy, suggests a manageable tolerability profile up to 5 years of follow-up.

Challenges and Trust in Telehealth Integration for Multiple Sclerosis Care: Enrique Alvarez, MD, PhD

The professor of neurology at University of Colorado School of Medicine talked about the challenges of telehealth and device-based data collection in clinical practice. [WATCH TIME: 4 minutes]

Friedreich Ataxia Therapy Nomlabofusp Increases Frataxin Levels in Phase 2 Study

Findings from a phase 2 study of nomlabofusp, a novel investigational therapy for Friedreich ataxia, demonstrated an increase in tissue frataxin levels and metabolic improvements.

Addressing Persistent Challenges in NMOSD Diagnosis and Treatment: Sumaira Ahmed

Despite advancements in NMOSD treatments, the founder and executive director of the Sumaira Foundation discussed how misdiagnosis, delayed diagnosis, and disparities in care remain significant challenges. [WATCH TIME: 4 minutes]

Short-Term Analysis Confirms Safety of Oral Edaravone in ALS Over 96 Weeks

Results from a recent phase 3 study presented at the 2025 MDA Conference reported that oral edaravone remained well tolerated in patients with ALS over 96 weeks, with no new safety concerns.

MDA Study Highlights Lack of Psychosocial Care Services for Duchenne Muscular Dystrophy

Despite facing mental health concerns with anger, aggression, or irritability, among others, slightly less than one-fourth of patients with DMD utilized psychosocial services such as counseling or therapy.

Sevasemten Reduces Muscle Injury Biomarkers of Becker Muscular Dystrophy in Latest Analysis

The phase 2 CANYON trial findings presented at the 2025 MDA conference highlight sevasemten’s potential in reducing muscle injury biomarkers in Becker muscular dystrophy.

Updates on Guidance for Diagnosing Pediatric and Late-Onset Multiple Sclerosis: Andrew Solomon, MD

The professor of neurological sciences at the University of Vermont discussed the work of the MS Differential Diagnosis Consortium, highlighting efforts to refine MS differential diagnosis and improve clinical accuracy. [WATCH TIME: 8 minutes]

Celebrating Milestones and Tackling New Frontiers in Pompe Disease: Priya Kishnani, MD

The professor of pediatrics and division chief of Medical Genetics at Duke University gave clinical insight on a presentation on the advancements and limitations of enzyme replacement therapy for Pompe Disease. [WATCH TIME: 3 minutes]

Episode 137: Advancing Neuromuscular Care and Research

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Robert Califf, MD. [LISTEN TIME: 9 minutes]

Gene Therapy RGX-202 Shows Positive Interim Results in Duchenne Muscular Dystrophy Trial

New biomarker data presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference of RGX-202 in the phase 1/2 trial highlight strong microdystrophin expression.

Using Baseline Paramagnetic Rim Lesion Count as Prognostic and Treatment Biomarkers in Multiple Sclerosis: Jiwon Oh, MD, PhD

The medical director of the Barlo Multiple Sclerosis Program at St Michaels Hospital discussed findings from phase 3 trials suggesting that baseline PRLs can predict disability accumulation and may serve as biomarkers for treatment response to tolebrutinib. [WATCH TIME: 6 minutes]

Givinostat Delays Duchenne Muscular Dystrophy Progression by 2 Years, Simulation Model Shows

A new analysis reported that givinostat, an oral histone deacetylase inhibitor recently approved for DMD, slowed disease progression by approximately 2 years compared with standard care.

Critical Lessons From Emergence of Enzyme Replacement Therapies in Pompe Disease: Priya Kishnani, MD

The professor of pediatrics and division chief of Medical Genetics at Duke University delved into the transformative impact of enzyme replacement therapy on Pompe disease, addressing its advancements, limitations, and promising innovations shaping its future. [WATCH TIME: 2 minutes]

Breaking Barriers: Donovan Decker’s Journey in Gene Therapy and Advocacy for LGMD

Donovan Decker, recipient of the 2025 MDA Legacy Award for Community Impact and Research, shared his powerful journey as a patient advocate and gene therapy pioneer, shedding light on the challenges and progress in LGMD.

Investigational Vatiquinone Slows Friedreich Ataxia Disease Progression Relative to FACOMS Natural History Cohort

Two separate long-term extension studies met their pre-specified endpoints, showing highly significant evidence of sustained treatment benefits in slowing disease progression in pediatric and adult patients.

SHIELD-DMD to Evaluate Therapeutic Potential of IL-6 Receptor Satralizumab in Duchenne Muscular Dystrophy

The design of a phase 2 trial to investigate the potential of satralizumab in boys with Duchenne muscular dystrophy was recently presented at the 2025 MDA Clinical & Scientific Conference.

Intrathecal Onasemnogene Abeparvovec Demonstrates Favorable Safety and Motor Function Stabilization in Treatment-Experienced Patients With SMA

The phase 3b STRENGTH study reported that a single dose of OAV101IT was well tolerated in treatment-experienced patients with spinal muscular atrophy, with motor function stabilizing over 52 weeks.

Long-Term Data Reinforces Safety of Gene Therapy Zolgensma for Spinal Muscular Atrophy

Real-world findings from the RESTORE registry presented at the 2025 MDA conference confirmed the established safety profile of onasemnogene abeparvovec over a 5-year period.

SAT-3247 and Restoring Muscle Regeneration in Duchenne Muscular Dystrophy: Phil Lambert, PhD

The chief scientific officer at Satellos Bioscience gave clinical insight on a new novel treatment and its mechanism of action in treating Duchenne muscular dystrophy. [WATCH TIME: 5 minutes]

NeuroVoices: Lily Sander on Advocacy, Community, and Raising Awareness for Charcot-Marie-Tooth Disease

Patient advocate and MDA Ambassador Lily Sander shared inspiring perspective on Charcot-Marie-Tooth disease, advocacy efforts, and the importance of connection

Early Trial Data Highlights Potential of Gene Therapy GNT0004 for Duchenne Muscular Dystrophy

Long-term follow-up data from an early-phase study of an AAV8-based gene therapy for DMD suggest the treatment was well-tolerated and provided sustained biochemical and functional benefits.

Mechanism and Promise Behind Del-Zota for Duchenne Muscular Dystrophy: Michael Flanagan, PhD

The chief scientific officer at Avidity Biosciences provided clinical perspective on the function and mechanism of del-zota, an investigational antisense treatment in development for DMD amenable to exon 44 skipping. [WATCH TIME: 3 minutes]

Ifetroban Shows Promise in Slowing Cardiac Decline in Duchenne Muscular Dystrophy

Findings from the phase 2 FIGHT DMD trial suggest an investigational oral thromboxane prostanoid receptor antagonist may preserve heart function in patients with DMD-associated cardiomyopathy.

New Post-Marketing Study to Test Omaveloxolone in Pregnant and Lactating Patients

This phase 4 study on omaveloxolone will assess long-term maternal and infant health, potentially informing future safety guidelines for patients with Friedreich ataxia.

Ocular Symptoms of Myasthenia Gravis Improved With Rozanolixizumab

The MyacarinG study post-hoc analysis demonstrated rozanolixizumab's potential to significantly reduce ocular symptoms in gMG, with consistent improvements across multiple scoring systems.

Dystrophin Expressing Chimeric Cell Therapy Demonstrates Long-Term Safety in Non-Ambulatory Duchenne Muscular Dystrophy

Three patients treated with DT-DEC01 showed improvements in various functional tests, including echocardiography, arm movements, grip strength, and spirometry after 12-24 months of treatment.

Baseline Characteristics and Updates for Phase 3 FORTIFY Study of BBP-418 in LGMD

In the study, most participants are aged 18-60, with 53% female and 73.1% carrying the c.826C>A mutation, while 15 patients are aged 12-18.