Marco Meglio

Andrew Lee, MD, PhD, cofounder and CEO of Spot Biosystems, discusses the company's extracellular vesicle platform, the first non-viral delivery of full-length dystrophin in patients with Duchenne muscular dystrophy.

Epilepsy expert Susan Duberstein, MD, explores how multidisciplinary neurocutaneous clinics improve diagnosis, surveillance, care coordination, and long-term outcomes for patients with complex neurocutaneous syndromes.

The submission is backed by phase 3 FoCus trial data showing ALXN1840 tripled copper mobilization and produced significant, sustained neurologic improvement compared with standard of care over 48 weeks.

Kristen Harris, MD, Medical Director of the Extended Recovery Unit at Hackensack Meridian JFK Johnson Rehabilitation Institute, discussed new findings on functional recovery and community discharge rates in acquired brain injury patients undergoing skilled nursing facility rehabilitation.

Ahead of World Brain Day, NeurologyLive explores 5 emerging strategies for preventing neurologic disease, from modifiable dementia risk factors to GLP-1 drugs, sleep science, the gut brain axis, and wearable technology.

A look at where things stand for Fragile X syndrome drug development, covering seven agents across four mechanisms as the field regroups from two recent Phase 3 setbacks.

Brett Morrison, MD, PhD, associate professor at Johns Hopkins University, recaps his PNS 2026 presentation on current and emerging biomarkers in peripheral nerve disease, from neurofilament light chain to muscle MRI and microRNA.

Laura Nisenbaum, PhD, Interim Chief Science Officer at the Alzheimer's Drug Discovery Foundation, shares her perspective on the FDA's approval of a subcutaneous starting dose for lecanemab and what it means for access, clinical practice, and the future of Alzheimer disease treatment.

The FDA granted Priority Review to zeleciment rostudirsen for exon 51-amenable Duchenne muscular dystrophy, with a PDUFA target action date of January 21, 2027.

The $100 million PROTECT-Cog trial will test whether adding a GLP-1 drug to the proven U.S. POINTER lifestyle intervention further cuts dementia risk in at-risk older adults.

PrevenTRON, a study of trontinemab, will enroll 1600 cognitively unimpaired participants identified by elevated plasma p-tau217, using time to clinical progression as the primary endpoint.

SAR448851 showed favorable safety, high CNS penetration, and up to 50% reductions in CSF soluble TREM2 in phase 1, supporting the design of TREMHANCE, a phase 2 trial now in final planning with p-tau217 as its primary endpoint.

The global phase 3 registration trial enrolled 1535 patients across 240 sites and has completed its 52-week double-blind treatment phase, with no unexpected safety signals observed, even in participants on concomitant antiamyloid therapies.

A cross-sectional survey of 4181 NARCOMS Registry participants found that only 7.4% had ever used semaglutide or tirzepatide, despite 40% meeting FDA-approved indications for the drugs.

Across 3 first-in-human studies in healthy volunteers and cognitively impaired adults, MK-2214 demonstrated dose-proportional pharmacokinetics, an approximately 80-day half-life, and near-complete suppression of free pS413 tau in CSF at doses of 20 mg and above.

Arif Dalvi, MD, director of the Comprehensive Movement Disorders Center at Palm Beach Health Network in Florida, discusses new Lancet Neurology data on staged bilateral focused ultrasound for Parkinson disease motor complications.

The 60 mg dose showed the largest treatment effect, slowing decline by 26% on CDR-SB, 42% on ADAS-Cog13, and 50% on MMSE at 18 months, while all doses produced 50% to 65% reductions in CSF total tau and unprecedented tau PET reductions across brain regions.

MCLENA-1 enrolled only 6 completers due to funding constraints but demonstrated 12 months of continuous lenalidomide was safe and tolerable, with nonsignificant trends favoring treatment on cognition and global outcomes.

The approval allows patients to begin lecanemab treatment at home via weekly subcutaneous injection, eliminating the 18-month IV requirement that previously preceded any at-home dosing.

The APPLAUDS trial will evaluate mivelsiran, an intrathecally delivered siRNA targeting amyloid precursor protein, in cognitively stable adults aged 40 to 55 with early-stage Down syndrome and early-stage Alzheimer disease who have elevated amyloid burden.

Leslie M. Thompson, PhD; Ravi Rajmohan, MD, and Jefferson W. Chen, MD, from UCI Health discuss the rationale, delivery, and early steps of REGEN4HD, the world's first neural stem cell phase 1b/2a clinical trial for Huntington disease.

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 RNA-targeted therapeutics

The director of the Division of Neuroscience at IRCCS Istituto Ortopedico Rizzoli discusses why targeting shared biologic pathways, rather than individual mutations, may represent a promising therapeutic strategy for Charcot-Marie-Tooth disease. [WATCH TIME: 2 minutes]

An Oxford study found that current smokers with MOGAD had a 2.2-times higher annualized relapse rate than never smokers, whereas past smokers showed no elevated risk, supporting smoking cessation as a modifiable management target.

Mind Moments®, a podcast from NeurologyLive®, brings you an exclusive interview with Daniel Ontaneda, MD, PhD. [LISTEN TIME: 20 minutes]

The first randomized, double-blind trial directly comparing the 2 anti-CD20 agents showed rituximab met the prespecified noninferiority criterion on MRI suppression, with a similar serious adverse event profile and a substantially lower cost.

Fariha Abbasi-Feinberg, MD, FAASM, FAAN, newly installed president of the American Academy of Sleep Medicine and medical director of sleep medicine at Millennium Physician Group, shares her clinical and policy priorities for the year ahead and her vision for the future of sleep medicine.

The professor of neurology and neuroscience at Johns Hopkins University School of Medicine discussed the ongoing debate around animal models in neuropathy research, the rise of iPSC-derived systems, and why he believes both approaches are needed going forward.

Cleveland Clinic pilots HAP-E, guiding older adults to manage epilepsy and cognitive decline with lifestyle tools, meds support, and virtual access.

Matthew Evans, BM, BCh, DPhil, a consultant neurologist at the University of Oxford, discusses the diagnostic approach to small fiber neuropathy and the challenges of confirming immune-mediated disease. [WATCH TIME: 3 minutes]