PhRMA Orphan Drug Report

ORPHAN DRUGS IN DEVELOPMENT FOR RARE DISEASES: More Than 400 Medicines in Development to Treat or Prevent Rare Diseases

AMERICA’S BIO PHARMACEUTICAL RESEARCH COMPANIES (PhRMA), February 2011

As we issue our report on rare diseases, I can’t help but think of the words of a mother whose daughter has Lennox-Gastaut Syndrome, which causes multiple,
daily seizures. In a documentary about the disease, she gives the following advice to other parents: “Don’t try looking at tomorrow. Don’t even think of the next hour. Just look for the next minute. And if the next minute is too far away, look for the next second. We’ve had to do this at times. But there is hope.”

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