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Delivering Protein Therapeutics to the CNS

An Orphan Drug receives an Orphan Drug Designation from the US FDA and the European Medical Agency. In the US, rare genetic diseases such as Tay-Sachs and Gaucher Disease are designated as Orphan Diseases because they have fewer than 200,000 patients in the US. The FDA created the Orphan Drug act in order to compel the pharmaceutical and biotechnology industries to develop drugs for small patient groups. Orphan Diseases and Orphan Drugs can be attributed to as few as 25 or 50 patients.