<code id='07B4C91591'></code><style id='07B4C91591'></style>
    • <acronym id='07B4C91591'></acronym>
      <center id='07B4C91591'><center id='07B4C91591'><tfoot id='07B4C91591'></tfoot></center><abbr id='07B4C91591'><dir id='07B4C91591'><tfoot id='07B4C91591'></tfoot><noframes id='07B4C91591'>

    • <optgroup id='07B4C91591'><strike id='07B4C91591'><sup id='07B4C91591'></sup></strike><code id='07B4C91591'></code></optgroup>
        1. <b id='07B4C91591'><label id='07B4C91591'><select id='07B4C91591'><dt id='07B4C91591'><span id='07B4C91591'></span></dt></select></label></b><u id='07B4C91591'></u>
          <i id='07B4C91591'><strike id='07B4C91591'><tt id='07B4C91591'><pre id='07B4C91591'></pre></tt></strike></i>

          00:00
          00:00 00:00 LIVE
          buffering
          Replay
          LIVE
          00:00 / 00:00
          LIVE
          CC
          Opacity :
          Share:
          Close

          comprehensive

          author:Wikipedia    - browse:1821
          Hiram Secrist (left), who has Duchenne muscular dystrophy, with his mother, Kristen Secrist (center), and his grandmother, Terrie Jordan. Courtesy Secrist family

          In mid-July, Kristen Secrist hopped on a call with her 5-year-old son’s doctor, who had urgent news: The first gene therapy had been approved for Duchenne muscular dystrophy. Her son, Hiram, would be a perfect candidate — if, she added, they could get him dosed in time.

          The treatment was approved only for 4- and 5-year-olds. Hiram turned 6 in three weeks. 

          advertisement

          “Oh, crap,” Secrist said to herself.

          Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

          GET STARTED Log In

          focus