Center for Rare, Undiagnosed and Genetic Diseases
Families with children suffering from rare diseases face a long and arduous journey from diagnosis to therapy. It is not unusual for this journey to take years. Sadly, patients without a diagnosis—more than 90% of patients with rare disorders—are offered nothing beyond supportive care.
Even with advances like genome sequencing, the diagnostic rate for rare diseases has remained stagnant. Rare diseases present challenges for clinical trials where most discoveries happen: small patient populations, young participants, or invasive therapies. Progress is slowed because clinicians and researchers are working independently rather than pooling the latest research and discovery.
The Center for Rare, Undiagnosed and Genetic Diseases will serve as a platform to bring forward a new approach to the diagnosis and study of rare diseases.
Donate to The Center for Rare, Undiagnosed and Genetic Diseases
Our Approach
The Center for Rare, Undiagnosed and Genetic Diseases aims to close the gaps in the diagnostic journey of rare disease patients by fostering collaboration between disciplines and investing in cutting-edge technology that will spur innovation. It will create connections between researchers and the patient community. Educational opportunities and a robust scientific infrastructure will equip researchers with the tools to make breakthroughs.
In addition, our unique partnership with WashU Medicine allows us to leverage our combined strengths, pipelines, and resources for the singular purpose of advancing rare disease patients from diagnosis to therapy.
The path forward
Your investment in the Center for Rare, Undiagnosed and Genetic Diseases will accelerate emerging research on rare childhood conditions, uncovering new therapies, treatments, and the answers so many families desperately seek.
| Center for rare, Undiagnosed and Genetic diseases | |
|---|---|
| Support for Personnel & Staffing Needs | $3.4 million |
| Establish Endowed Scientist Position | $2 million |
| Expand Program Implementation | $3.4 million |
| TOTAL | $8.8 million |