News

The POLARIS program includes 3 ongoing phase 1/2 clinical trials investigating the efficacy and safety of investigational gene therapy EXT101 in patients with SCN1A postive Dravet syndrome.

In honor of Purple Day®, held on March 26, 2026, Mary Anne Meskis, chief executive officer of the Dravet Syndrome Foundation, highlighted ongoing efforts and future steps to raise awareness for rare epileptic disorders.

The founder and CEO at the Hereditary Neuropathy Foundation discussed how learning about patient experiences has influenced research in Charcot-Marie-Tooth disease. [WATCH TIME: 5 minutes]

Apply for a $40,000 international MS research award honoring senior women scientists advancing care for women; submissions close June 12, 2026.

The FDA granted accelerated approval to tividenofusp alfa for Hunter syndrome, introducing the first enzyme replacement therapy designed to cross the blood-brain barrier and target neurologic disease.

The chief medical officer at Edgewise discussed long-term MESA data showing sustained functional stabilization with sevasemten in Becker muscular dystrophy and its potential as a disease-modifying therapy.

Experts discussed new biomarker data from a phase 1/2 study, presented at the 2026 MDA Conference, of the gene therapy SGT-003 in boys living with Duchenne muscular dystrophy.

Jonathan Charlesworth, PhD, chief scientific officer at Noctrix Health, discusses emerging research connecting restless legs syndrome with neurologic conditions such as multiple sclerosis, and the role of the FDA-cleared Nidra wearable device in patient care.

The 2026 MDA National Ambassador discussed the importance of connecting patients’ experiences with scientific understanding, as well as fostering a sense of community among those living with neuromuscular disease. [WATCH TIME: 2 minutes]

The division chief of neuroimmunology at Brigham and Women’s Hospital provided clinical insights on a recently published study in JAMA Neurology covering EBNA-1 antibodies as a diagnostic clue in neuroinflammatory diseases like multiple sclerosis. [WATCH TIME: 4 minutes]

In a plain language summary of the ADHERE trial, subcutaneous efgartigimod improved disability and reduced relapse risk by 61% in CIDP, with sustained benefit and a favorable safety profile.

The assistant professor of medicine at the University of Toronto discussed population-level evidence suggesting MS may precede EBV infection in rare cases and its implications for disease pathogenesis and diagnosis.

A large JAMA Neurology study found that persistently elevated EBNA-1 antibody titers across serial samples strongly differentiated multiple sclerosis from MOGAD and NMOSD, supporting its role as an adjunct diagnostic biomarker.

New data from the pivotal study of investigational therapy zilganersen in Alexander disease will be presented at the 2026 American Academy of Neurology (AAN) annual meeting in Chicago.

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

A new retrospective analysis presented at AD/PD 2026 showed that cutaneous phosphorylated α-synuclein testing reduced diagnostic and management-related health care expenditures.

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 chronic traumatic encephalopathy!

In honor of Down Syndrome Awareness Day, neurologist Elizabeth Head, PhD, discusses neurologic comorbidities, biomarker advances, and emerging precision medicine approaches in Down syndrome–associated Alzheimer disease.

Neurology News Network for the week ending March 21, 2026. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 20, 2026.

Sleep medicine specialist Karin Johnson, MD, FAAN, FAASM, shared the main takeaways from a recently published guideline on evaluating and managing obstructive sleep apnea in hospitalized adults.

Levacetylleucine met its primary end point in a phase 3 trial of patients living with ataxia-telangiectasia, with no drug-related serious adverse events reported, supporting the sNDA.

An analysis of baseline data from the phase 2b LUMA study suggested that keystroke dynamics captured through a smartphone-based typing task could provide a reliable method for assessing disease progression in Parkinson disease.

From FDA regulatory milestones for investigational agents to emerging clinical data across sleep disorders, this roundup highlights notable developments shaping the evolving landscape of orexin-targeted therapies.

The research portfolio director at the Muscular Dystrophy Association highlighted the collaborative spirit, the expanding therapeutic pipeline, and the growing emphasis on patients’ voices in neuromuscular disease on display at MDA 2026. [WATCH TIME: 8 minutes]

Early ASPIRO trial data show autologous stem-cell dopamine precursors stay safe at 12 months, with improved PD motor scores and PET-confirmed grafting.

MRI-guided focused ultrasound steers IV gene therapy to mouse brains, lowering cortical tau—though hippocampal response stays absent.

Phase 1 data suggest plasma GFAP levels may correlate with cognitive outcomes in patients with Alzheimer disease treated with troculeucel, an investigational autologous NK-cell therapy.