<code id='9FFA1497B9'></code><style id='9FFA1497B9'></style>
    • <acronym id='9FFA1497B9'></acronym>
      <center id='9FFA1497B9'><center id='9FFA1497B9'><tfoot id='9FFA1497B9'></tfoot></center><abbr id='9FFA1497B9'><dir id='9FFA1497B9'><tfoot id='9FFA1497B9'></tfoot><noframes id='9FFA1497B9'>

    • <optgroup id='9FFA1497B9'><strike id='9FFA1497B9'><sup id='9FFA1497B9'></sup></strike><code id='9FFA1497B9'></code></optgroup>
        1. <b id='9FFA1497B9'><label id='9FFA1497B9'><select id='9FFA1497B9'><dt id='9FFA1497B9'><span id='9FFA1497B9'></span></dt></select></label></b><u id='9FFA1497B9'></u>
          <i id='9FFA1497B9'><strike id='9FFA1497B9'><tt id='9FFA1497B9'><pre id='9FFA1497B9'></pre></tt></strike></i>

          knowledge

          knowledge

          author:comprehensive    Page View:7
          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

          comprehensive

          Novavax promises a turnaround & Lilly roils the obesity market
          Novavax promises a turnaround & Lilly roils the obesity market

          SammyKimballforSTATCanNovavaxfinallygetitright?What’sa“triple-G”drug?AndisNovoNordisklosingground?We

          read more
          Physicians and social media: If you see something, say something?
          Physicians and social media: If you see something, say something?

          AfterJoannaGaines,co-hostofHGTV's"FixerUpper,"shownherewithherhusband,Chip,postedanultrasoundofherba

          read more
          Medicare proposal to cover medical devices draws a mixed reaction
          Medicare proposal to cover medical devices draws a mixed reaction

          AdobeThemedicaldeviceindustryissplitonwhetheraproposednewMedicarereimbursementpathwaywilldoanythingt

          read more

          App could help diagnose ear infections more accurately — and at home

          AUWSchoolofMedicineprofessorpracticesusingtheappandfunneltocheckhisdaughter'sear.DennisWise/Universi