How one family gave a critical boost to gene therapy research

A family affected by Sanfilippo syndrome raised millions of dollars for research. The Food and Drug Administration just approved a gene therapy the support helped make possible.

<img src='https://npr.brightspotcdn.com/dims3/default/strip/false/crop/1378x1120+0+0/resize/1378x1120!/quality/85/format/webp/?url=http%3A%2F%2Fnpr-brightspot.s3.amazonaws.com%2Ff2%2Fae%2F660d57f84079b118bb3a7ea0cd5d%2Foneill-family-credit-robert-clarke-photography-2.jpg' alt='Glenn (from left), Beckham, Cara and Eliza O'Neill pose for a family photo in 2015. Eliza has Sanfilippo syndrome, a rare inherited disease.'/><p>A family affected by Sanfilippo syndrome raised millions of dollars for research. The Food and Drug Administration just approved a gene therapy the support helped make possible.</p><p>(Image credit: Robert Clark)</p><img src='https://media.npr.org/include/images/tracking/npr-rss-pixel.png?story=nx-s1-6014739' />