The company has decided to pause enrollment in the high-dose cohort of its phase 1 clinical trial for NGN-401 after a participant experienced a serious treatment-related adverse event.

The phase 2 trial is expected to include 80 adults with genetically confirmed CMT who will be followed for a 21-day treatment period, using changes in functional assessments as the primary outcome.

The SKYCLARYS PASS registry, an international observational cohort study, aims to include approximately 300 omaveloxolone-naïve patients participating in the Friedreich’s Ataxia Global Consortium's UNIFIED natural history study.

The change in opinion was after reconsidering the totality of data from the phase 3 Clarity AD trial, a large-scale study of lecanemab featuring more than 1500 patients with early-stage Alzheimer disease.

Investigators are set to test the effects of 300 mg/day fasudil after the therapy showed promising data on ALS-related biomarkers in 180 mg/day doses.

Here's the latest multidisciplinary multiple sclerosis (MS) research from the International Journal of MS Care.

Here's some of what is coming soon to NeurologyLive® this week.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is on Friedreich ataxia.

According to the company, VQ-101 is the first small molecule to show over 75% activation of the lysosomal enzyme GCase in humans, with phase 1 results in healthy volunteers indicating tolerability and exposure levels that support once-daily dosing.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending November 15, 2024.

After just 6 weeks of treatment, patients showed antibody levels 16 times higher than those in the placebo group, highlighting the vaccine’s potential to slow disease progression.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Lara Jehi, MD, MHCDS. [LISTEN TIME: 20 minutes]

Initial findings showed significant improvements from baseline in the disease-specific, patient-reported quality of life scores in both the high and low dose AAV-GAD groups, with no significant change in the sham group at 26 weeks.

In a previously completed phase 1 study, the treatment resulted in significant improvements in wakefulness at doses of 1.0 mg and 2.5 mg, prompting a follow-up study.

The software, designed using thousands of brain MRI scans, is used for automated detection and severity grading of ARIA-E and ARIA-H for safety monitoring of new Alzheimer disease therapies.

Nerivio, a nondrug option for acute migraine attacks and prevention of migraine with or without aura, has been FDA-approved for several years in adolescents aged 12 and older as well as adults.

NGN-401, an AAV9 gene therapy in development for Rett syndrome, was considered well-tolerated, with treated patients showing consistent gains in skill and developmental milestones.

A previous 5-year analysis showed that pediatric patients with aromatic L-amino acid decarboxylase deficiency when treated with eladocagene exuparvovec experienced durable developmental, motor, and cognitive improvements.

In a longitudinal study lasting 2 years, impaired test performance in semantic fluency and congruent speed were observed in patients with MOGAD relative to healthy controls.

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine discussed the newly updated multiple sclerosis criteria, highlighting their focus on probabilistic assessment, advanced technology integration, and early intervention to improve patient care.

Neal K. Shah, CEO at CareYaya Health Technologies, shared his perspective on how the new administration could unleash innovation in neurotech, reduce regulatory barriers with Elon Musk’s involvement, and accelerate innovations that could improve neurological care for those with Alzheimer and dementia.

Capsida’s CAP-002 gene therapy, currently advancing through IND-enabling studies, aims to address the unmet needs of patients with STXBP1-related developmental and epileptic encephalopathy by stably supplementing STXBP1 protein.

The phase 1 study, which is currently recruiting, includes a 4-week screening period, a 52-week treatment period, and a 4-year long-term extension to continue to test the effects of HG202.

Italo Linfante, MD, director of interventional neuroradiology at Baptist Health Miami Neuroscience Institute, discussed the importance of brain aneurysms in the context of neurology.