Structural Connectome Alterations in Alternating Hemiplegia of Childhood.
Brain imaging reveals specific patterns in a rare neurological disorder, offering measurable markers to track treatment progress in future trials.
Structural connectome analysis of Alternating Hemiplegia of Childhood patients reveals widespread white matter alterations and disrupted motor and cerebellar network connectivity in this ultra-rare ATP1A3-related disorder. These neuroimaging biomarkers provide novel, objective metrics for disease characterization that may serve as endpoints in future clinical trials for this condition with no approved disease-modifying therapy.
What the study was
- Study design
- observational neuroimaging study
- Population
- Alternating Hemiplegia of Childhood patients
- Category
- Diagnostics
- Maturity
- Exploratory
- Journal
- Brain topography
Why it surfaced
First structural connectome characterization in AHC (ultra-rare ATP1A3 disorder); neuroimaging biomarkers could support clinical trial endpoint development; high unmet need with no approved disease-modifying therapy.
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