Welcome To IRDiRC
Our vision: Enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention
Goal 1
All patients coming to medical attention with a suspected rare disease will be diagnosed within one year if their disorder is known in the medical literature; all currently undiagnosable individuals will enter a globally coordinated diagnostic and research pipeline
Goal 2
1000 new therapies for rare diseases will be approved, the majority of which will focus on diseases without approved options
Goal 3
Methodologies will be developed to assess the impact of diagnoses and therapies on rare disease patients
Latest News
Global Genes – 2025 Rare Drug Development Symposium
Global Genes, IRDiRC Patient Advocacy Constituent Committee member organizes its 2025 RARE Drug…
The Wiedemann-Steiner Syndrome Grant Program 2025
The Wiedemann-Steiner Syndrome Foundation, in partnership with the Orphan Disease Center University of…
NIH: Request for Information for Better Tools to Predict Toxicities Resulting from Oligonucleotide Therapeutics
The National Center for Advancing Translational Sciences (NCATS) is seeking input from the…