Marco Meglio

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 parasomnias.

Subgroup analyses revealed stronger efficacy signals in biomarker-defined groups, particularly in Caucasian participants with measurable baseline myostatin levels.

These results highlight the age range and ambulation status for which viltolarsen is a key treatment option for DMD patients amenable to exon 53 skipping.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Andy Berkowski, MD, PhD. [LISTEN TIME: 26 minutes]

Applied Therapeutics noted that it is reviewing the FDA's feedback and will request a meeting to discuss requirements for either a resubmission of the NDA or appeal of the agency's decision.

Over the 6-month treatment period, patients saw improvements in cataplexy frequency, excessive daytime sleepiness, cognition, and work productivity with AXS-12.

AL002 did not meet the primary endpoint of slowing Alzheimer's clinical progression, as measured by the Clinical Dementia Rating Sum of Boxes and showed no treatment effects on secondary clinical and functional endpoints.

Over 1 year of treatment, simufilam failed to distinguish itself from placebo on the primary end points of Alzheimer’s Disease Assessment Scale-Cognitive Subscale 12 and Alzheimer’s Disease Cooperative Study-Activities of Daily Living.

In a 76-patient cohort, HL192 was safe and well tolerated across 5 observed dose levels, with a treatment-emergent adverse event incidence that mirrored placebo.

The study, presented at the 2024 AANEM) Annual Meeting, evaluated 52 patients who underwent surgery for carpal tunnel syndrome and explored the comparative effectiveness of the Conway and Zeidman scales in predicting patient outcomes.

Hearing loss, as defined by objective audiograms, was associated with an additional risk of developing PD later in life in a dose-dependent manner.

Amanda Hare, DNP, a doctor of nursing practice specialized in movement disorders, gave comments on the innovation behind the StrivePD app and how it can revolutionize personalized treatment management for patients with Parkinson disease.

The multicenter, 24-week study will feature 20 patients with MG, testing changes on MG-ADL as well as several other secondary outcomes, including patient-reported assessments.

Overall, subcutaneous efgartigimod was safe and effective in patients with seropositive MG, demonstrated by consistent improvements in Myasthenia Gravis-Activites of Daily Living.

The trial, a 20-week study assessing lamotrigine in DLB, will use change in Clinical Dementia Rating-Sum of Boxes as the primary efficacy end point, with several other notable secondary outcomes.

For more than a 2-year treatment period, 95% of patients reported taking at least 95% of their daily medications with zilucoplan.

Over a 12-month treatment period, patients on deferiprone demonstrated decreased blood ferritin and hippocampal QSM but caused accelerated cognitive decline and increased regional brain atrophy.

The findings suggest that future phase 3 trials should explore the duration of pain relief and headache recurrence, with the goal of developing structured treatment guidelines for PTH.

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 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 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.

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.

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]