Phase 1 safety established through prior clinical use for repurposed drugs in these specific indications.
Vascular malformations and thrombotic vasculopathies affecting CNS function, including conditions with seizure and stroke manifestations.
Histiocytic disorders with central nervous system involvement, focusing on inflammatory and infiltrative pathologies.
Lysosomal storage diseases and proteinopathies with progressive neurological decline.
Ion channel disorders and neuromuscular transmission defects affecting motor function and paroxysmal neurological symptoms.
