Latest Conference Coverage

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.

Over the 5-year period, treatment-related adverse events with the gene therapy were mild or moderate, with most occurring within the first 90 days after infusion.

The associate researcher at the University of California, San Francisco discussed a refined EAE model that could better replicate MS pathology, enabling deeper insights into demyelination, neuronal loss, and remyelination. [WATCH TIME: 6 minutes]

The professor of neurology and neurosurgery at McGill University discussed the evolving understanding of neuroimmune interactions in MS, highlighting how these interactions contribute to disease progression and potential repair mechanisms. [WATCH TIME: 4 minutes]

The assistant professor in the department of neurology at Mount Sinai talked about a recent study that highlighted how socioeconomic status and diet could significantly impact physical and cognitive outcomes in patients with MS. [WATCH TIME: 2 minutes]

The professor of neurology at University of Colorado School of Medicine discussed the evolving landscape of MS treatment, highlighting the role of personalized medicine and biomarker-driven decision-making. [WATCH TIME: 5 minutes]

The instructor in the department of radiology at Weill Cornell Medicine discussed how structural and functional brain connectomes can potentially improve predictions of MS progression and treatment response. [WATCH TIME: 4 minutes]

A post-hoc analysis of patients from the CHAMPION-NMOSD trial revealed that the majority received their initial meningococcal vaccination within 6-months of their last rituximab dose.

The professor of neurosurgery and physiology at University of Colorado School of Medicine talked about how vagus nerve stimulation may promote remyelination and functional recovery in multiple sclerosis. [WATCH TIME: 4 minutes]

A late-breaking study presented at the 2025 ACTRIMS Forum reported that a modified Atkins diet reduced inflammation and altered immune cell metabolism in multiple sclerosis.

At the 2025 ACTRIMS Forum, the director of the MS Comprehensive Care Center at Stony Brook Medicine discussed the evolution of high-efficacy treatments for MS. [WATCH TIME: 4 minutes]

Data from more than 16,000 trial participants point to possible challenges with assessing and safely administering novel therapeutics among those with comorbidities—which may be present in as much as half of the patient population.

In the CAVS-MS study, 19% of participants with atypical or radiological-only presentations met the 2024 McDonald Criteria for MS, enabling earlier treatment initiation.

GFAP changes positively correlated with gadolinium-enhancing lesion volume (P = 0.18) and negatively with left ventricular volume (P = -0.19).

The vice president and senior global program lead for Hematology and Cell Therapy at BMS gave clinical commentary on encouraging phase 1 findings of CD19-XT in progressive and relapsing multiple sclerosis.

In both HERCULES and GEMINI trials, tolebrutinib reduced the risk of 6-month confirmed disability worsening, especially in participants with at least 4 baseline paramagnetic rim lesions.

The pediatric neurologist at Children’s Hospital of Philadelphia gave perspective on some of the main issues the clinical community is figuring out with gene therapies and their integration to clinical practice. [WATCH TIME: 3 minutes]

Glatiramer acetate depot treatment showed stability in EDSS scores over 3 years, suggesting halted progression in patients with primary progressive MS.

At the 2025 ACTRIMS Forum, the system chief of neuroimmunology and multiple sclerosis at OhioHealth discussed the need for better tools to detect MS progression. [WATCH TIME: 3 minutes]

The director of the MS and Demyelinating Disease Center at Texas Tech University Health Sciences Center talked about early diagnosis, timely treatment, and minimizing therapy transition gaps to prevent severe disability in patients with NMOSD. [WATCH TIME: 4 minutes]

After 24 months after switching to cladribine, 100% of patients with relapsing MS remained free from T1 gadolinium-enhancing lesions, with no reported cases of PML or rebound disease activity.