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Meeting a Gene Therapy Success Story

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Girl with her parents as two adults look on

Gene therapy patient Evelyn Villarreal, 3, along with her parents Milan and Elena. I shared the story of her successful treatment for spinal muscular atrophy at a Senate HELP Committee hearing on NIH science and stewardship on Aug. 23, 2018. NICHD Director Diana Bianchi and NCI Director Ned Sharpless are in the foreground. Credit: C-SPAN


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