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5%
There are approximately 7,000 rare diseases, yet only five percent have FDA-approved treatments.1
5+
On average, it takes more than five years to obtain a rare disease diagnosis due to limited professional knowledge of the signs and symptoms.1
40%
40% of people living with rare disease are misdiagnosed at least once.2
Therapeutic Areas
These are the conditions for which we have an approved therapy or a research and development program.
References
1. Global Genes
2. EURORDIS
