Isabella Ciccone, MPH

The chief executive officer and founder at CureDuchenne discussed efforts to bring specialized physical therapy and infrastructure support to underserved regions to improve care for patients impacted by Duchenne worldwide. [WATCH TIME: 5 minutes]

Treatment with DYNE-251 revealed dose dependent exon skipping and dystrophin expression as well as improvement in multiple functional end points in both cohorts of the phase 1/2 DELIVER trial.

Based on the positive topline data, the company plans to submit for regulatory approval for a higher dose of nusinersen for spinal muscular atrophy treatment.

Treatment with vutrisiran was associated with benefits across multiple well-established clinical measures of disease progression compared with the placebo in patients with transthyretin-mediated amyloidosis with cardiomyopathy.

Jim Cassidy, MD, PhD, chief medical officer at SpringWorks Therapeutics, talked about how the prioritization of mirdametinib, a promising treatment for both adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas.

NeuroQuant previously received clearance from the FDA as a medical device intended for automatic labeling, visualization, and volumetric quantification of segmentable brain structures in MRI analysis.

The application is based on results of Prilenia Therapeutics’ phase 3 study that assessed the safety and efficacy of pridopidine in patients with Huntington disease.

The system is composed of the Neuroblade, a single-use multifunctional neuroendoscope; the Neuropad, a reusable medical-grade tablet; and Clearpath, a disposable transparent access sheath.

For the majority of patients who were either aquaporin-4-antibody seropositive or seronegative, rituximab was the first disease-modifying therapy administered for neuromyelitis optica spectrum disorder.

Mirdametinib, a treatment in development for adult and pediatric patients with neurofibromatosis type 1-associated plexiform neurofibromas, was granted a PDUFA target action date of February 28, 2025.

The FDA has granted fast track designation to [18F]PI-2620 for 3 neurodegenerative conditions, highlighting its potential to improve early diagnosis and treatment.

The submission was based on results from the phase 3 Vivacity-MG3 study, which demonstrated nipocalimab’s superiority over placebo when added to standard of care in patients with generalized myasthenia gravis.

Findings from a recent study analyzing 3 cohorts suggested that certain proteins in the cerebrospinal fluid may have potential as biomarkers or therapeutic targets in progressive supranuclear palsy.

The chief executive officer and chief medical officer at Quince Therapeutics talked about recent findings from the phase 3 ATTeST study assessing EryDex in pediatric patients with ataxia telangiectasia.

A recent cohort study reported that use of enzyme-inducing antiseizure medications alongside direct-acting oral anticoagulants was not associated with a difference in risk of thromboembolic events.

IMU-838 daily doses of 30-mg and 45-mg suppressed the development of gadolinium-enhancing lesions by 78% and 74% compared with the placebo at 24 weeks in relapsing-remitting multiple sclerosis.

William Hu, MD, PhD, FAAN, a neurologist at RWJBarnabas Health’s Robert Wood Johnson University Hospital in New Brunswick, NJ, shared new advances to detect and treat Alzheimer disease.

EryDex did not meet its primary end point possibly because of delays in treatment reducing the number of patients who received the agent, and different treatment effects based on age.

DT-216P2 shows promise in treating patients with Friedreich ataxia based on positive phase 1 trial results that indicated a significant increase in frataxin mRNA levels.

The director of academic clinical research at Orlando Health reflected on the journey of a blood test for traumatic brain injury to clinical use, highlighting the collaborative efforts and patient participation that made it possible. [WATCH TIME: 3 minutes]

PTC Therapeutics has plans to submit a new drug application for vatiquinone, a small molecule inhibitor of 15-lipoxygenase in development for patients with Friedreich ataxia, in late 2024.

The director of academic clinical research at Orlando Health talked about applying Abbott's blood test for suspected mild traumatic brain injury, providing results within 15 minutes, in the clinical practice. [WATCH TIME: 5 minutes]

Patients with myelin oligodendrocyte glycoprotein IgG-associated disease with severer attacks had higher C5b-9 levels than those with milder attacks.

A recent review showed different fluid biomarkers were involved in neurodegeneration, oxidative stress, metabolism, microRNA and novel genes in spinocerebellar ataxia type 3.

The postdoctoral researcher at Amsterdam University Medical Center talked about the development of the DAAE score, a tool for predicting the risk of transition to secondary progressive multiple sclerosis. [WATCH TIME: 8 minutes]

In prior research, findings have shown that losartan reduced the risk of epilepsy in patients by blocking astrocyte activation and reducing blood brain barrier damage.

Research indicated that up to 46% of patients diagnosed with presumed autoimmune limbic encephalitis tested negative for all currently identified central nervous system antigens.

Researchers detected annual changes in patients with preataxic and early ataxic spinocerebellar ataxia in brain MRI imaging, clinical scores, gait parameters, and retinal thickness.

The professor of human genetics at the University of Miami discussed the significance of various genetic factors in Alzheimer risk and highlighted ongoing research, therapeutic challenges as well as the need for global collaboration. [WATCH TIME: 10 minutes]

Recent findings showed that treatment with satralizumab was likely associated with a reduction in the concomitant use of immunosuppressive therapies in neuromyelitis optica spectrum disorder.