New data from the HELIOS trial shows AMX0035 improves glycemic control and stabilizes symptoms in Wolfram syndrome, paving the way for future studies.

FDA approves study to test Capsida's CAP-002 gene therapy for STXBP1-DEE, offering hope for improved seizure control and developmental outcomes in affected patients.

The director of the Tuberous Sclerosis Program at Cleveland Clinic provided clinical commentary on a novel clustering analysis in TSC, revealing how phenotype-genotype insights can lead to more personalized treatment and surveillance.

Committee chairs Vincent Timmerman, PhD, MSc, and Kathrin Doppler, MD, provide a preview of the 2025 PNS Annual Meeting, giving clinicians an inside look at what to expect from the upcoming conference.

Phase 1b data show oral microbiome therapy MaaT033 slows ALS progression and is well tolerated, supporting future phase 2 development efforts.

A recent interim analysis of a 5-year observational study showed sustained benefit of pitolisant in reducing excessive daytime sleepiness and cataplexy in patients with narcolepsy.

MeiraGTx's AAV-GAD gene therapy receives FDA RMAT designation, promising innovative treatment for Parkinson's disease through targeted brain infusion.

A review highlighted the role of serum neurofilament light chain as a potential biomarker in MOG antibody-associated disease, with findings pointing to associations with attack severity.

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

A recently published population-based study reported that patients with MS or NMOSD had a significantly higher risk of autoimmune rheumatic diseases than matched controls.

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 B-cell depleting medications for MS.

The phase 2 MAGNIFY trial demonstrated that zervimesine (CT1812) slowed lesion growth in patients with geographic atrophy secondary to dry AMD, showing a 28.6% reduction over 18 months.

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending May 9, 2025.

Catch up on any of the neurology news headlines you may have missed over the course of April 2025, compiled all into one place by the NeurologyLive® team.

Over long-term treatment, fenfluramine significantly reduced seizure frequency, increased seizure-free days, and improved quality of life in patients with Dravet syndrome.

A study involving 20 patients with ALS found that longitudinal muscle MRI detected progressive atrophy and correlated with muscle strength loss, supporting its potential as a biomarker for ALS progression and clinical trials.

Evan L. Smith, PhD, assistant professor of Physical Medicine & Rehabilitation at University of Michigan, discussed how emotional wellbeing, motivation, and evidence-based therapies can significantly influence outcomes in MS.

Leveraging the Enroll-HD dataset, recent data revealed consistent links between antidopaminergic medication use and worsened cognitive and motor outcomes in Huntington disease.

Early findings from the ASPIRO trial showed ANPD001 was safe, well-tolerated, and led to marked improvements in motor symptoms and daily functioning.

STN-FUS provided more sustained tremor reduction, improved bradykinesia, and had a higher responder rate at 12 months, though both treatments showed comparable reductions in rigidity and motor scores.

A 17-year-old with NMOSD and rituximab treatment developed PML after several relapses and secondary hypogammaglobulinemia, leading to his death 3 months after diagnosis.

The research portfolio director at the Muscular Dystrophy Association highlighted advances in gene-targeted therapies and biomarker development as potential key drivers of progress in ALS.

Theranica's FDA-cleared, drug-free migraine device gains expanded access, more than doubling coverage to 80 million Americans as young as 8 years old who are diagnosed with migraine.

Sunny Brous, a patient advocate living with ALS, shared how her decade-long journey with the disease shaped her mission to build connection, advocate for change, and empower the ALS community.

Baron, a neurologist and headache specialist at Cleveland Clinic, explained the significance, clinical data, and patient potential behind CT-132, the first FDA-approved digital therapeutic for migraine prevention.