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REPORTAmerica's biopharmaceutical research companies are developing 566 medicines for patients with rare diseases.
This report outlines the list of Medicines in Development for rare diseases and includes the product name, sponsor, official FDA designation and development status.
There is an urgent need for continued research to map the genes responsible for rare diseases, identify genetic and other biologic anomalies that contribute to these conditions, and understand the natural history of rare diseases to inform the development of potential therapies.