News

The phase 2 DIMENSION study results showed that SAGE-71 was well-tolerated but did not meet its primary end point, leading to Sage Therapeutics’ decision to discontinue its development.

At week 10, investigators observed no differences between triheptanoin and placebo on the frequency of movement disorder events in patients with Glut1 deficiency disorder.

The dean and chief academic officer at the University of Miami Miller School of Medicine discussed the university’s investment in computational biology to advance aging and neuroscience research. [WATCH TIME: 5 minutes]

The associate professor of neurology at Georgetown University provided clinical insight on the origins of nilotinib and its medicinal promise in treating patients with Dementia with Lewy bodies, a neurodegenerative disorder.

The global, placebo-controlled, 12-week study aims to enroll 330 participants and expects topline results in the first half of 2026.

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 professor in residence at UCLA Health discussed the critical considerations when deciding appropriate patients for gene therapies, specifically AAV vectors, in treating muscular dystrophies. [WATCH TIME: 4 minutes]

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 professor in residence at UCLA Health provided clinical insight on the limitations of certain gene therapy vehicles for muscular dystrophies, and the need for better vectors that are more myotropic. [WATCH TIME: 3 minutes]

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.

The William S. and Lois Stiles Edgerly Professor of Neurology at Yale School of Medicine talked about the ongoing evolution of multiple sclerosis diagnostic criteria and the importance of comprehensive testing to guide early intervention and improve outcomes. [WATCH TIME: 5 minutes]

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.

A safety analysis of the phase 3 MOXIe Part 2 trial (NCT0225435) highlighted the frequency and timing of TEAEs with omaveloxolone (Skyclarys; Biogen) treatment.

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.

Neurology News Network. for the week ending November 9, 2024. [WATCH TIME: 4 minutes]

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.

The doctor of nursing practice specialized in movement disorders discussed specific data the StrivePD app captures and how it can enhance quality of life for patients with Parkinson disease. [WATCH TIME: 3 minutes]

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.