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Gene therapy developer uniQure said it will submit a marketing application for accelerated approval for investigational AMT-130 for Huntington’s disease after the FDA reversed its position and agreed that a 3-year analysis of a phase I/II study showing benefit was acceptable.
The FDA also will reconsider clemidsogene lanparvovec — an investigational gene therapy for mucopolysaccharidosis II, or Hunter syndrome, that it rejected in February — for accelerated approval using existing clinical data, Regenxbio announced.
High-dose supplements of docosahexaenoic acid (DHA), an omega-3 fatty acid, penetrated the brain in a placebo-controlled trial of people at risk for dementia but led to no differences in cognitive performance or brain volume over 24 months. (eBioMedicine)
Rotating waves appeared to coordinate information flow across sensory and motor systems in a mouse study. (Science)
One in five patients with a type 1b spontaneous spinal cerebrospinal fluid leak had rare functional FBN2 variants, an exome sequencing study found. (Lancet Neurology)
A history of child abuse was linked with a higher long-term risk of migraine in men but not women, a 20-year longitudinal cohort study showed. (Headache)
The “forever chemical” perfluorohexanesulfonic acid was associated with multiple sclerosis in two U.S. cohorts. (Environmental Research)
The parents of a 2-year-old girl involved in a drowning incident on Memorial Day sued to prevent Texas Children’s Hospital from testing her for brain death. (Texas Tribune)
A within-participant comparison showed that the investigational PET tracer MK-6240 detected early tau pathology in Alzheimer’s disease more often than the approved agent flortaucipir (Tauvid). (The Lancet)
In a single-arm trial, unilateral magnetic resonance-guided focused ultrasound ablation was safe and effective for Parkinson’s motor complications, but bilateral treatment led to small motor gains and persistent moderate or severe adverse events. (Lancet Neurology)
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Source link : https://www.medpagetoday.com/neurology/generalneurology/121885
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Publish date : 2026-06-23 18:14:00
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