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New Insights into Batten Disease CLN1 from a Patient Facing Registry

2025-09-26 neurology Title + abstract only
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There is currently no FDA approved treatment for Batten disease CLN1, a rapidly progressive, fatal, and rare pediatric neurodegenerative disease. In this article, we aimed to collect retrospective clinical data from the families to identify CLN1 patients for a future patient facing natural history study and clinical trial. We have therefore developed and launched an institutional review board approved Batten disease CLN1 registry (https://cln1registry.collaborationspharma.com). We implemented a ...

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