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
Congrats to all those involved.
Nice to hear these success stories.