Many rare disease patients use drugs off-label based on limited data because they have no better options available. In fact, it is estimated that 95 percent of rare diseases do not yet have a single FDA-approved treatment . One of the authors of this
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Read the complete post at http://healthaffairs.org/blog/2017/03/21/for-rare-disease-patients-a-pathway-to-hundreds-of-new-therapies/
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Mar 21 2017, 08:46 AM
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