Marco Meglio

The senior vice president of medical and clinical affairs for Avadel Pharmaceuticals provided perspective on a recently published analysis demonstrating the benefits of once-nightly sodium oxybate in narcolepsy type 1 and type 2.

Investigators observed no new safety concerns, no cases of Alzheimer-related imaging abnormalities, and no notable changes in laboratory evaluations and EKG.

Steve Hughes, MD, chief medical officer at Avidity Biosciences, provided commentary on recently announced positive topline data for AOC 1044, an investigational agent for Duchenne muscular dystrophy.

Take a look at 5 of the most-anticipated clinical trial data readouts expected in 2024 that neurology health care professionals should keep their eyes on.

Mind Moments®, a podcast from NeurologyLive®, brings you a review of 2023, with insights from Sharon Cohen, MD, FRCPC; Robert A. Hauser, MD, MBA; Alberto Espay, MD, PhD; Carolina Ferreira Atuesta, MD, MSc; and Natalie Goedeker, CPNP. [LISTEN TIME: minutes]

The neurologist at Cleveland Clinic shared thoughts on a recently published study assessing the relationship between blood biomarkers and change in cognitive function and brain volumes in a cohort of professional fighters.

The research aimed to assess FMD prevalence and clinical manifestations in both elderly and younger onset individuals, revealing insights into symptom variations across different age groups.

The newly announced trial, dubbed ReTain, will featured 500 individuals with preclinical Alzheimer disease who will be randomized to ACI-35.030 or placebo for a maximum of 4 years.

The senior vice president, global head of medical affairs, and chief medical officer at Acadia Pharmaceutical, gave an overview of LILAC-2, a long-term extension study assessing trofinetide (Daybue), the first approved therapy for Rett syndrome.

Elizabeth M. Cassidy, MPH, research project manager at the TSC Alliance, provided clarity on a presentation at AES 2023 examining the association between TSC-associated neuropsychiatric disorders and seizure duration.

In a phase 3 study, patients on omaveloxolone had less physical impairment compared with patients who received placebo, as measured by the modified Friedreich Ataxia Rating Scale.

A-synuclein-positive athletes were similar to α-synuclein-negative athletes on demographics and other serum and blood biomarkers; however, these patients had lower grey matter volumes in the right inferior orbitofrontal, right anterior insula, and right olfactory cortices.

Subgroups of narcolepsy types 1 and 2 had significant reductions in a number of secondary outcomes, including number of sleep stage transitions, number of nocturnal arousals, and improvements in sleep quality.

AOC 1044 was well-tolerated in healthy volunteers, delivering dose-dependent increases in PMO concentrations in skeletal muscle following administration.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Jennifer Gudeman, PharmD. [LISTEN TIME: 17 minutes]

The positive data report could potentially build on phase 2 study of Duchenne muscular dystrophy in which CAP-1002 met its primary end point.

Lobule VIIB showed a non-linear pattern of lower volume with each Hoehn and Yahr-increment bilaterally, with the most significant group differences at stages 4-5 compared with controls.

The chief medical officer of Biohaven discussed early-stage data supporting the efficacy and safety of BHV-7000, a selective Kv7.2/7.3 potassium channel activator in development for patients with epilepsy.

Also known as ATH-1017, fosgonimeton demonstrated a safe profile, with directional improvements in cognitive, functional, and biomarker assessments.

The OneRF Ablation System follows the Evo cortical and sEEG electrode product lines, marking a significant step in NeuroOne's journey to offer innovative solutions for neurological conditions.

The frequent use of REN wearable device for the acute treatment of migraine was shown to reduce the number of monthly treatments in adolescents, with a similar reduction in migraine and headache days seen in a pivotal prevention trial in adults.

Maura McCarthy, head of corporate development at Skyhawk Therapeutics, provided comment on a new phase 1 study assessing SKY-0515, a small molecule candidate as a potential treatment for Huntington disease.

Pitolisant demonstrated a dose-dependent improvement in excessive daytime sleepiness and fatigue, with a safety profile that was consistent with previous studies. Additional data to be shared in early 2024.

While specialists still debate the diagnostics of impaired cognition, the identification of patients at the earliest stages is beginning to be met through new biomarkers and available assays.

Using outcomes such as the CDR-SB, ADAS-Cog12, MMSE, and ADCOMS, patients treated with NE3017 for 6 months demonstrated equal, if not greater, improvements than traditional antiamyloid therapies at 18 months.

Over a 32-week treatment period, patients on trofinetide continued to show improvements on the primary efficacy outcomes of Rett Syndrome Behavior Questionnaire and Clinical Global Impression-Improvement total scores.

The findings further supported the idea that prevention and assessment of cardiovascular risk in patients with narcolepsy is critical to ongoing care.

Results showed that evobrutinib did not produce a more superior reduction in annualized relapse rates than teriflunomide, a previously approved disease-modifying agent.

Although not powered for statistical significance, results showed improvements in ALS Functional Rating Scale and slow vital capacity after treatment with PrimeC.

The vice president of the Rare Disease Translational Center at the Jackson Laboratory provided context on a recently published study suggesting restoration of stathmin-2 as a therapeutic approach for ALS.