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A Phase 1-2 trial of AAV9-GLB1 gene therapy in children with Type II GM1 gangliosidosis demonstrates significant biochemical improvements and stabilization of neurodevelopmental decline, marking a pivotal step toward treating this fatal lys
The 12-month BOND study found that high-dose benfotiamine (600 mg daily) did not significantly improve corneal nerve fiber length or other neurophysiological markers in patients with type 2 diabetes and symptomatic polyneuropathy, despite s
This phase 1-2 trial demonstrates that delpacibart etedesiran (del-desiran), an innovative antibody-oligonucleotide conjugate, effectively reduces toxic DMPK mRNA and ameliorates missplicing in patients with Myotonic Dystrophy Type 1, marki
A multi-cohort study demonstrates that women with amyloid-β pathology show higher plasma p-tau217 levels and faster tau PET accumulation than men, providing a biological basis for their increased risk of rapid cognitive decline in preclinic
Five-year data from the ULTIMATE I and II open-label extension demonstrate that ublituximab maintains exceptionally low relapse rates and slows disability progression in relapsing multiple sclerosis, emphasizing the clinical superiority of
A five-year prospective study reveals that 13.6% of stroke patients with mild cognitive impairment progress to major VCI, identifying age, diabetes, atrial fibrillation, and small vessel disease as key predictors for long-term cognitive dec
The FASTEST trial demonstrated that while recombinant factor VIIa administered within two hours of intracerebral haemorrhage onset significantly reduced hematoma growth, it failed to improve functional outcomes at 180 days and increased the
The TRACE-5 trial demonstrates that tenecteplase significantly improves functional outcomes for patients with basilar artery occlusion within 24 hours of onset, offering a safe and effective extension of the therapeutic window for this high
A large-scale matched cohort study from the UK Biobank identifies in-hospital delirium as a sentinel marker for long-term multisystem vulnerability. Associated with increased risks of sepsis, AKI, and falls, delirium signals a profound phys
This systematic review confirms that Neurofilament Light Chain (NfL) in blood and CSF effectively distinguishes behavioral variant frontotemporal dementia from psychiatric disorders, offering a powerful tool for early and accurate diagnosis
A comprehensive study explores how polygenic risk scores for late-onset Alzheimer’s disease (LOAD) influence early-onset (EOAD) cases. While these scores do not independently predict EOAD onset or cognitive decline, they reveal significant
A large-scale longitudinal study reveals that over 80% of community-dwelling older adults harbor mixed neuropathologies, identifying five distinct profiles that dictate specific cognitive decline trajectories, with ADNC and LATE-NC clusters
This population-based cohort study highlights how cumulative psychosocial stress across the life course significantly escalates the risk of neurovascular and neurodegenerative diseases, identifying depression as a critical target for interv
The integrated analysis of the VALOR trial and its open-label extension reveals that early initiation of tofersen significantly slows functional decline, preserves respiratory capacity, and improves survival in patients with SOD1-ALS over a
This randomized clinical trial demonstrates that real acupuncture significantly reduces migraine frequency and disability compared to sham acupuncture. Crucially, baseline brain connectivity patterns within the default mode and subcortical
A landmark autopsy study reveals that Chronic Traumatic Encephalopathy (CTE) involves extensive spinal cord pathology, including tau, TDP-43, and amyloid-β deposits. These findings suggest that repetitive head impacts trigger a widespread e
A prospective study of over 130,000 adults reveals that consuming 2-3 cups of caffeinated coffee or 1-2 cups of tea daily significantly reduces dementia risk and cognitive decline, whereas decaffeinated coffee shows no protective effect.
A landmark study using CDC ADDM Network data reveals significantly elevated mortality risks for young adults with autism, intellectual disability, and cerebral palsy, highlighting a critical need for improved transition-of-care strategies a
A large multicenter real-world study indicates that cenobamate provides superior effectiveness and seizure freedom rates compared to brivaracetam, lacosamide, and perampanel in adults with drug-resistant focal epilepsy, despite a higher inc
The OPTION trial reveals that tenecteplase administered 4.5 to 24 hours post-stroke in patients with non-large vessel occlusion and salvageable tissue significantly improves functional recovery, although it carries a higher risk of symptoma
