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FDA speeds innovation in rare disease therapies

from U.S. Food and Drug Administration

Patients often need advocates, and that can be especially true for people with a rare disease, who have unique problems and may have little or no support or available treatment. The Food and Drug Administration is committed to helping patients and advancing rare disease therapies through the development of "orphan" medical products, including drugs, biologics (such as a protein, vaccine or blood product), and devices used to treat a rare disease or condition. The Orphan Drug Act defines a disease as rare if fewer than 200,000 people in the United States have it. more


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