LRRK2 kinase inhibition to normalize lysosomal trafficking in Parkinson's disease

🧪 Hypothesis Details

LRRK2 G2019S mutations cause autosomal dominant PD through hyperactivation that impairs vesicular trafficking via Rab GTPase phosphorylation. Multiple Phase 1 programs (DNL151, BIIB094) have demonstrated target engagement (>90% Rab10 dephosphorylation) with manageable safety profiles. Lung toxicity (lamellar body accumulation) represents a dose-limiting concern requiring intermittent dosing strategies. The biomarker (Rab10 phosphorylation) enables patient selection and response monitoring.

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↻ Rerun processing chain

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