The National Institutes of Health, U.S. Food Drug Administration, and the
Foundation for the Institutes of Health unveiled a public-private partnership Wednesday aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease.
“They’ve waited a long time for something to be focused this way to address the incredibly wrenching stories we see all around us of children and adults with rare diseases where we can do a diagnostic test to tell them what they have but beyond that haven’t had much to offer,” Francis Collins, the pioneering genetics researcher and longtime NIH director, told STAT.
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