Mapping Clinical Progression to Brain Atrophy in CLN2 Patients Under Cerliponase Alfa Treatment: A Prospective Neuroimaging Study - PubMed
3 hours ago
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- #biomarkers
- #CLN2 disease
- The study examined the link between brain atrophy and clinical progression in CLN2 patients treated with cerliponase alfa.
- Longitudinal MRI and clinical assessments showed significant cortical thinning, with a 4.2% annual loss compared to 12.5% in natural history controls.
- Reductions in global cortical thickness over 24 weeks were associated with changes in motor, language, and movement disorder scales.
- Multivariate analysis identified a latent dimension linking regional brain atrophy to worse clinical outcomes, accounting for 82% of shared variance.
- Brain atrophy patterns were connected to network architecture, suggesting MRI morphometry as a biomarker for CLN2 disease monitoring and therapy guidance.