Marco Meglio

The committee all agreed that tofersen presents a favorable risk-benefit profile in this particularly challenging patient population.

After results indicated significant slowing of ALS disease progression, Coya noted it plans to file an IND with the FDA in the second half of 2023 for a potential clinical study thereafter.

If approved, ABBV-951 will become the first subcutaneous delivery of carbidopa/levodopa, often considered the standard of care for symptom management for patients with Parkinson disease.

The professor of neurology and pediatrics at University of Rochester Medical Center shared her thoughts on the 2023 MDA Clinical and Scientific Conference, and the progress made in the treatment of neuromuscular disorders.

Efgartigimod-treated patients consistently outperformed placebo regardless of concomitant medication, thymectomy status, and timing of diagnosis.

After previously showing positive effects on MG-ADL and QMG, ravulizumab continued to show improvements in ocular and respiratory muscle domains in myasthenia gravis.

In a phase 3 study, tofersen failed to meet its primary end point of functional change, but did meet its target engagement of SOD1 protein and reductions in neurofilament light.

More than 90% of patients, ambulatory and non-ambulatory, experienced improvements in Medical Research Council score within 6 months of treatment.

Mechanistically, reboxetine and a combination of reboxetine and oxybutynin improved pharyngeal collapsibility and respiratory control.

Ahead of the 2023 MDA Clinical and Scientific Conference, Anne Connolly, MD, provided perspective on conversations surrounding holistic approaches to neuromuscular diseases.

Frailty domains such as exhaustion, slow gait speed, low grip strength, and low physical activity were all associated with incident Parkinson disease.

Michael Dwyer, PhD, director of IT and Neuroinformatics Development at the Buffalo Neuroimaging Analysis Center, provided insight on how artificial intelligence techniques may be used to monitor disease progression in multiple sclerosis.

The director of functional neurosurgery at Baptist Health Miami Neuroscience Institute provided perspective on the growth of high intensity focused ultrasound and the lessons learned when treating essential tremor.

Plasma P-tau217 was associated with cognitive decline across several cognitive tests in 2 different cohorts and with conversion to AD dementia in the large-scale, longitudinal BioFINDER-1 cohort.

Originally approved under the accelerated approval pathway, a final decision on a traditional approval for lecanemab is expected to come in early July.

Findings showed that global cognition declines in the prediagnostic period in men but not women with incident Parkinson disease, further indicating cognitive heterogeneity in the prodromal period by sex.

Among countries with non-zero mechanical thrombectomy access, there was a 460-fold difference in availability of this care between the country with the highest and lowest access rate reported.

Early data has shown a substantial neuroprotective benefit in response to c-Abl inhibition by IkT-148009.

On PET imaging, amyloid continued to accumulate over time, with participants in the solanezumab and placebo groups showing similar increases over time.

The first and only approved CGRP nasal spray for acute migraine is expected to be available in pharmacies in July 2023.

Treatment with a lower dose of CNM-Au8 resulted in significantly decreased risk of death, feeding tube placement, assisted ventilation, and all-cause hospitalization, among other survival outcomes.

Batoclimab, a fully human anti-FcRn monoclonal antibody, has demonstrated an ability to significantly alleviate patients’ symptoms and improve quality of life in myasthenia gravis.

Compared with healthy controls, those with isolated RBD performed significantly worse on all cognitive assessments except in tasks of executive function.

The director of IT and Neuroinformatics Development at the Buffalo Neuroimaging Analysis Center provided perspective on the sudden explosion of artificial intelligence, and how it can be applied to MS care.

Efgartigimod resulted in rapid impact on health-related quality of life, with improvements that were consistent across multiple measures and were similar for the effect seen across 2 treatment cycles.

The effect of cognitive rehabilitation on personalized cognitive goals was specifically found 6 months after treatment completion, whereas the benefits on patient-reported cognitive complains did not persist.

Supplementary data from the phase 3 Clarity AD study served as the basis for the lecanemab’s new review, for which a decision is expected to come by July 6, 2023.

Taylor Gonyou, DO, a multiple sclerosis fellow at Michigan Institute for Neurologic Disorders, detailed a proteomic biomarker panel that gauges multiple sclerosis disease activity, with potential to impact clinical decision-making.

Now 4 years after the approvals of the first calcitonin gene-related peptide inhibitors, new therapies are being developed to address the lingering needs of patients with refractory migraine.

Between the 2 therapies, ozanimod was associated with significantly lower risks of safety outcomes, including adverse events leading to discontinuation, herpetic infections, bradycardia, and abnormal liver enzymes.