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
Fondazione Telethon and Orchard Therapeutics complete transfer of marketing authorization of Strimvelis for ADA-SCID in Europe
Following a positive opinion from the European Medicines Agency (EMA), the transfer of…
Defining rare conditions in the era of personalized medicine
Don’t miss out the new IRDiRC publication developed by Therapies Scientific Committee (TSC)…
New Publication: Making Rare Disease Research Attractive to Companies by IRDiRC Chrysalis Task Force
Accessible at https://doi.org/10.1177/26330040231188979, the Chrysalis Task Force identified key financial and non-financial factors…