New Hope for Rare Disease Patients

Rare disease isn’t as rare as it sounds. Ten percent of Americans have a rare disease– and there are almost 7,000 known rare diseases.  A rare disease, also known as an orphan disease, is defined as a disease that affects fewer than 200,000 people in the US.  In recent years, the biopharmaceutical industry has made great strides in the research and treatment of rare diseases. The FDA has approved more than 770 medications to treat rare diseases since the Orphan Drug Act was passed in 1983.  In recent years, new treatments have been developed to treat such rare diseases as cystic fibrosis, blood cancers, Gaucher disease, hereditary angioedema, hypophosphatasia, lysosomal acid lipase deficiency, and neuroblastoma. With ongoing research and new technologies, the 30 million Americans suffering from rare disease have more hope for the future than ever before.

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