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Story summary
- GRIN-related neurodevelopmental disorder (GRIN-NDD), caused by NMDA receptor dysfunction, currently lacks therapies addressing its underlying biology.
- Radiprodil, previously tested for neuropathic pain, has been repositioned for GRIN-NDD.
- Its advancement to a global Phase 3 trial within four years shows how coordinated capital, scientific expertise, and regulatory strategy can speed development.
- The case highlights the need for sustained investment in patient identification and trial readiness to overcome challenges in ultra-rare conditions.
