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reportMedicines in Development: Rare Diseases
A Report on Orphan Medicines in the Pipeline
Today, there are more than 700 medicines in development for rare diseases affecting 30 million Americans. A new report from PhRMA explores these medicines that target these diseases and conditions.
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Related Resources
Medicines in Development for Rare Diseases 2021 Drug List
Dive deeper into the full list of more than 700 medicines currently in development targeting many of the approximately 8,000 known rare diseases.