Neurology news.

The FDA approved Zanvastro (zilganersen) for pediatric and adult patients with Alexander disease. Evidence included a randomized controlled study of 49 patients aged 2 years and older and an open-label substudy of four younger children; reported motor benefits supported approval.

An observational computational study identified approximately 1.5 million phenotype-associated brain cells from 584 donors with Alzheimer’s-related phenotypes. The PASCode framework linked cell subpopulations and gene-expression patterns to pathology, cognition and depression, with findings validated in external datasets.

A case-control study across 17 international cohorts compared MRI data from 369 females with bulimia nervosa and 417 healthy controls. Bulimia nervosa was associated with modest reductions in nucleus accumbens volume and temporal cortical surface area, while binge-eating frequency correlated with lower surface area in several regions.

EMA’s human medicines committee recommended 12 medicines for approval at its 14–17 September 2026 meeting, including two biosimilars and two generics. It also recommended extensions of therapeutic indications for 11 medicines.

The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), the first approved treatment for pediatric Sanfilippo syndrome type A. An open-label, single-arm study reported maintained or improved cognition compared with historical controls; the announcement provided no sample size or numerical effect estimates.

A prospective multicenter cross-sectional study of 681 participants evaluated a five-class visual reading framework across four tau PET tracers. The framework showed high reader agreement and classified more cognitively unimpaired participants and those with mild cognitive impairment as tau-positive than the regulatory-approved binary approach.

A JAMA Neurology review emphasizes that HINTS is most effective in patients with continuous dizziness and spontaneous nystagmus at rest. The authors advise screening for central features first and warn that inappropriate patient selection can contribute to missed strokes and unnecessary imaging.

A randomized trial of 703 older adults with mild cognitive impairment and hearing loss found no significant reduction in dementia-level impairment with hearing aids over 24 months. A prespecified secondary analysis found more cognitive improvement with hearing aids than with hearing care education.

A JAMA Neurology review summarizes MRI features and lesion evolution in myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). It highlights differences from multiple sclerosis and suggests surveillance MRI has less clinical utility because asymptomatic radiological activity is rare.

The FDA approved Emcitate (tiratricol) as the first FDA-approved therapy for peripheral thyrotoxicosis in MCT8 deficiency. Evidence came from a randomized, placebo-controlled trial and a longer-term open-label study involving infants through adults; the announcement did not report sample sizes or numerical treatment effects.

A prospective single-center registry of 4,221 atrial fibrillation ablation procedures found higher 30-day stroke/TIA rates with pulsed field than radiofrequency ablation: 0.47% versus 0.10%. The association persisted after propensity score weighting, but only 12 events occurred and the observational design limits causal interpretation.

All newborns in Sweden will now be screened for metachromatic leukodystrophy following a recommendation from Socialstyrelsen. The programme aims to identify affected children before symptoms and irreversible neurological damage develop, allowing timely consideration of stem cell-based gene therapy.

An observational analysis compared structural MRI data from 45,900 controls and 2,698 people with neurological, psychiatric or addiction-related conditions. Alzheimer’s disease and mild cognitive impairment showed the strongest associations with older predicted brain age; ADHD and autism showed no significant overall difference from controls.