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Latest orphan diseases Stories

2014-06-23 04:20:31

LONDON, June 23, 2014 /PRNewswire/ -- Highlights: - Creation of an international CRO with complete service offering from Phase I to Phase IV post-authorisation studies - Appointment of a highly experienced Board, including Professor Trevor Jones CBE, Chairman, Ronald Openshaw, Chief Executive Officer and Professor Sir Christopher Evans, Non-Executive Director - Completed with GBP12.5m of funds raised from Welsh...

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...

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...


Word of the Day
penuche
  • A fudgelike confection of brown sugar, cream or milk, and chopped nuts.
'Penuche' is a variant of 'panocha,' a coarse grade of sugar made in Mexico. 'Panocha' probably comes from the Spanish 'panoja, panocha,' ear of grain.
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