Latest Conference Coverage

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]

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.

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]

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.

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.

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.

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]

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]

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

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]

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.

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]

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.

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.

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.

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.

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.

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]

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

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.

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]

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.

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

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

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.

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.

A recent study shows that eteplirsen, a drug promoting dystrophin production, significantly slowed the decline in heart function, specifically left ventricular ejection fraction, in patients with Duchenne muscular dystrophy.

Spanning 14 weeks, the study evaluates age-appropriate dosing, safety, pharmacokinetics, and clinical effects through once-weekly injections of efgartigimod PH20 SC in juvenile myasthenia gravis.