“The State-of-the-Art of N-of-1 Therapies and the IRDiRC N-of-1 Development Roadmap” is now published in Nature Reviews Drug Discovery.
This exciting review explores the emerging field of N-of-1 genetic therapies, specifically designed for individuals with rare diseases caused by unique genetic variants. This pioneering approach represents a major step toward tailored treatments for ultra-rare conditions. The publication covers:
- The concept and potential of N-of-1 therapies for individualized treatment of rare diseases
- Real-world cases where successful therapies have been developed
- Regulatory, practical, and ethical challenges impacting access and development
- A roadmap for the future of N-of-1 therapy development
? Read the full article here: https://doi.org/10.1038/s41573-024-01059-3
More information about the N-of-1 Task Force: https://irdirc.org/preparing-for-genetic-n-of-1-treatments-of-patients-with-ultra-rare-mutations/