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Last updated on April 17, 2014 at 11:16 EDT

Latest orphan diseases Stories

2014-01-08 08:28:14

LA JOLLA, Calif., Jan. 8, 2014 /PRNewswire/ -- Auspex Pharmaceuticals, Inc., a Phase 3 clinical stage biopharmaceutical company focused on developing and commercializing novel medicines for the treatment of orphan diseases, today announced the completion of two separate financings totaling $35 million and appointment of a new Board member: -- $20 million Series E equity financing led by Deerfield Management Company and supported by existing investors, Thomas, McNerney and...

2013-10-17 08:29:37

Seasoned Industry Veterans Driving Auspex's SD-809 Through Phase 3 Development for the Treatment of Chorea Associated with Huntington's Disease LA JOLLA, Calif., Oct. 17, 2013 /PRNewswire/ -- Auspex Pharmaceuticals, Inc., a privately-held clinical stage biopharmaceutical company focused on developing innovative therapeutics for the treatment of orphan diseases, today announced the appointment of four life science industry veterans to the company's executive team: -- Pratik Shah, Ph.D.,...

2011-07-26 11:54:00

PHILADELPHIA, July 26, 2011 /PRNewswire-USNewswire/ -- The Raymond and Ruth Perelman School of Medicine at the University of Pennsylvania announces the launching of a first-of-its-kind interdisciplinary center focused on discovering novel treatments for orphan diseases. The Center will bring together, without institutional walls, all necessary approaches to attacking and treating orphan diseases: establishing dedicated research support facilities, translating findings into therapies,...

2011-06-09 23:29:26

Mutations in genes essential to survival are behind so-called orphan diseases, explaining in part why these diseases are rare and often deadly, according to a study appearing in The American Journal of Human Genetics. The new finding contrasts sharply with what is known about mutations in non-essential genes being the drivers of common diseases having higher prevalence rates, according to scientists at Cincinnati Children's Hospital Medical Center who conducted the research. The...