Dr. Aartsma-Rus is a professor in translational genetic and a member of IRDiRC Therapies Scientific Committee. In this video she tells us why IRDiRC is needed in the rare disease field and why it is important have different stakeholders collaborating. IRDiRC does it and all its members are committed to reach the new IRDiRC goals for 2027:
- Develop the means to diagnose patients with suspected rare diseases within one year of coming to medical attention
- Develop 1,000 new therapies for rare diseases and particularly for diseases with no approved treatment
- Create methods for assessing the impact of diagnoses and therapies on rare disease patients