Marco Meglio

A coalition of Charcot-Marie Tooth patient groups, clinicians, and pharma companies has published the first consensus framework for designing clinical trials in the disease.

The FDA has cleared Roche's Elecsys pTau217, a blood test that uses a single biomarker to both rule in and rule out amyloid pathology in adults being evaluated for Alzheimer disease, across both primary and specialty care settings.

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 Huntington disease!

AMO Pharma reached agreement with the FDA, UK MHRA, and Health Canada on the design of a registrational study of AMO-02 for congenital myotonic dystrophy type 1, which will use hospitalization as its primary outcome measure.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Pezhman Roohani, MD. [LISTEN TIME: 17 minutes]

Full results from the phase 3 TEMPO-2 trial, published in Lancet Neurology, showed that flexible-dose tavapadon significantly improved motor symptoms and daily function in people with early Parkinson disease.

David Stamler, MD, Chief Executive Officer of Alterity Therapeutics, discusses the FDA's alignment on the pivotal Phase 3 program for ATH434 in multiple system atrophy and what a successful trial could mean for a disease with no approved disease-modifying therapies.

The cerebrovascular neurosurgeon at University Medical Center New Orleans discussed performing the first THUNDERBOLT stroke thrombectomy in the United States and what computer-assisted vacuum thrombectomy offers over traditional aspiration.

Oak Hill Bio announced dosing of the first participant in BEACON, a pivotal phase 3 trial evaluating rugonersen, an antisense oligonucleotide originally developed by Roche, for Angelman syndrome.

The new data from ALKIVIA marks the first phase 3 study to show statistically significant, clinically meaningful improvement in immune-mediated necrotizing myopathy, a subtype with no approved therapy.

A phase 3 trial found that solriamfetol significantly improved wakefulness and reduced excessive daytime sleepiness in Chinese patients with obstructive sleep apnea, with a safety profile consistent with prior studies conducted in Western populations.

A large claims-based cohort study found that two doses of the recombinant zoster vaccine reduced the risk of shingles by 64% to 81% in adults with multiple sclerosis, a population with elevated shingles risk and historically low vaccine uptake.

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 acute ischemic stroke!

A large multicenter study found that APOE genotype and a polygenic Alzheimer risk score together predict up to a 10-year difference in disease onset among people carrying an extra copy of the APP gene, whether through an APP duplication or Down syndrome.

The FDA approved TAUKLARIFY (florquinitau F 18 injection), a tau PET imaging agent, for identifying tau neurofibrillary tangle pathology in adults being evaluated for Alzheimer disease.

The FDA has cleared Quantum BioPharma's investigational new drug application for Lucid-MS, allowing the company to begin a phase 2 trial of the first-in-class demyelination-targeting compound in progressive multiple sclerosis.

CurePSP and UCSF announced enrollment of the first participant in the PSP Trial Platform, believed to be the first platform trial ever conducted specifically for progressive supranuclear palsy, designed to test multiple investigational drugs simultaneously.

Ractigen Therapeutics announced completion of enrollment and first dosing across all cohorts of its phase 2 trial of RAG-17, an investigational siRNA therapy for SOD1-mutated ALS, building on phase 1 data recently published in Nature Medicine.

Horacio Kaufmann, MD, Director of the Dysautonomia Center at NYU Langone, discusses the prospective validation of prodromal MSA criteria, how olfactory testing can sharpen specificity, and how close the field is to biomarker-driven early diagnosis.

Annexon reported that all 10 patients in the first US and European cohort of its open-label FORWARD study showed rapid, clinically meaningful improvement in strength within days of a single tanruprubart infusion for Guillain-Barré syndrome.

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 Spinal Muscular Atrophy!

Investigators independently tracked the 6 patients treated in a Parkinson disease gene therapy trial that was abandoned mid-study after its sponsor became insolvent, finding a reassuring safety profile and mixed signals of motor benefit through up to 3 years.

The FDA has granted 510(k) clearance to VoxNeuro's Cognitive Function Neuroimaging software, an EEG/ERP-based tool designed to give clinicians an objective, adjunctive measure of cognitive function.

Six FDA PDUFA decisions this fall could reshape care in Alzheimer disease, DMD, Sanfilippo syndrome, Alexander disease, and SMA, here's what clinicians need to know about the data behind each.

The FDA has approved oveporexton, marketed as Orzeyful, as the first therapy to directly restore orexin signaling and address the full symptom range of narcolepsy type 1.

The associate professor at the University of Michigan covered the 2026 Peripheral Nerve Society Annual Meeting, highlighting emerging themes in GBS, SMA, TTR neuropathy, RFC1, and what is still keeping the field up at night.

The FDA has granted Regenerative Medicine Advanced Therapy designation to sasineprocel, an autologous cell therapy for Parkinson disease, based on early data from the ongoing ASPIRO trial.

The FDA has accepted Saol Therapeutics' resubmitted NDA for SL1009 in pyruvate dehydrogenase complex deficiency, setting a new PDUFA target action date of December 30, 2026.

A new cross-sectional study found that professional identity partially explains the connection between burnout and missed nursing care among neuro-oncology nurses in China, with psychological capital buffering both pathways.

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 chronobiology in neurology!