PhRMA reports record number of rare disease drug candidates under development
This article was originally published in Scrip
As patient groups, researchers and health organisations across the globe mark 28 February as World Rare Disease Day, the Pharmaceutical Research and Manufacturers of America (PhRMA) is reporting that a record 460 compounds are in mid-to-late clinical development to treat rare or orphan conditions, such as Gaucher's disease, Duchenne muscular dystrophy (DMD) and Pompe's disease.
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