Marco Meglio

Recruitment has begun for Part C of the study, with topline data from this part of the trial expected to report in Q2 2025.

Patients with CIDP treated with riliprubart reported improved quality-of-life and fatigue measures, as well as reduced neurofilament light levels.

The data, which comes less than a month after the FDA granted RMAT designation, highlighted the impacts of AMT-130 on disease progression in Huntington disease.

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Most treatment-emergent adverse events for patients on UB-312 were mild or moderate, and comparable to placebo.

Overall, treated patients showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed volumes.

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ALX-001, a first-in-class compound, will be assessed in an additional cohort of patients with Alzheimer disease as the company continues to plan for phase 2 studies.

After 48 weeks of continued treatment, those in the high-dose frexalimab group showed a 41% reduction in neurofilament light.

Data from the large-scale, phase 3 OCARINA 2 trial was the basis for the approval.

Over the 3-year treatment period, buntanetap resulted in improvements in motor and nonmotor functions in patients with postural instability and gait difficulty, a group with faster disease progression.

The neurologist at Baptist Health provided context on identifying treatment approaches for varying degrees of migraine, the need to optimize medication use, and when patients may be experiencing more than just migraine.

in both the overall population and a per-procotol population, PrimeC-treated individuals experienced improvements on ALS Functional Rating Scale and increased survival compared with placebo.

Following the 12-week program, participants reported numerous physical and neuropsychiatric benefits in their daily living, such as increased energy level, better mood, and improvement in balance.

The newly added cohort allows for greater opportunity to treat patients earlier, with the hope of impacting disease course and preserving muscle.

Suvecaltamide did not meet the primary endpoint in the phase 2b trial but showed numeric improvements and was well-tolerated.

Vitrisiran demonstrated a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in both the overall population and those who received a monotherapy of vutrisiran without tafamidis.

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After following patients for more than 10 years, arrhythmias were considered the most important cardiac predictor of survival, with progressive neurologic disability and diabetes mellitus identified as further major contributors.

Overall, the treatment resulted in dose-dependent trends of improvement on key clinical measures including Total Functional Capacity and Composite Unified Hungtinton's Disease Rating Scale.

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Clinically meaningful reductions in seizures through fenfluramine is likely to contribute to nonseizure benefits such as improvement in comorbidities, decreased use of other antiseizure medications, and reduction in premature death.

Istradefylline led to significant reductions in tremor and motor symptoms in patients with Parkinson disease over 24 weeks.

Bezisterim has shown potential in improving non-motor symptoms like fatigue and sleep issues, with promising results from previous studies.

Modified Functional Status Questionnaire, an assessment of activities of daily living, includes several different notable domains, including quality of interaction, social activity, and psychological function.

Although the data used predated 2019, istradefylline was associated with a statistically significant lower odds of the overall incidence of treatment-emergent adverse events than other adjunctive therapies for Parkinson disease.

In a large-scale analysis. 70% of patients with essential tremor and 70% of undiagnosed patients reported dissatisfaction with the therapeutic options available to treat their condition.

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Patients treated with at least 12.5 mg/day of oral prednisone for at least 3 months had an 88% reduction in the risk of relapse compared with those who did not receive this regimen.

The therapy is the first and only neonatal Fc receptor blocker approved for the treatment of CIDP, and will be available as a once-weekly, 30- to 90-second subcutaneous injection.