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Raring to Go: Companies Race to Develop Rare Disease Medicines

By Peter Winter

Monday, October 15, 2012

Small patient populations have suddenly become big business for biotechnology and pharmaceutical companies alike. The pipeline is rapidly filling with candidate medicines targeting rare and ultra-rare diseases. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), last year biopharmaceutical research companies had approximately 460 medicines targeting rare diseases either in clinical trials or awaiting FDA review.

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