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2-year-old battles rare disease

2014-03-08 07:49:24| Industrial Machines - Topix.net

An Omaha boy is one of only 2,000 in the world with a rare disorder called Hunter syndrome, and with little research available, his family is leading the charge to find out what might stop it.

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NIH Scientists Pursue New Therapies To Improve Rare Disease Drug Development

2013-10-02 06:18:51| drugdiscoveryonline News Articles

Four new pre-clinical drug development projects at the National Institutes of Health will target a form of blindness and diseases characterized by cardiac problems. The projects were selected for their potential to treat specific rare diseases and to help scientists uncover new information that can be shared with other researchers

Tags: development improve drug disease

 
 

MassBio panel tackles why rare disease treatments cost so much

2013-03-17 19:52:04| Biotech - Topix.net

David Meeker, right, CEO of Genzyme Corp., speaks at a panel on pricing of rare disease therapies at the MassBio annual meeting.

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