<code id='0DD551B837'></code><style id='0DD551B837'></style>
    • <acronym id='0DD551B837'></acronym>
      <center id='0DD551B837'><center id='0DD551B837'><tfoot id='0DD551B837'></tfoot></center><abbr id='0DD551B837'><dir id='0DD551B837'><tfoot id='0DD551B837'></tfoot><noframes id='0DD551B837'>

    • <optgroup id='0DD551B837'><strike id='0DD551B837'><sup id='0DD551B837'></sup></strike><code id='0DD551B837'></code></optgroup>
        1. <b id='0DD551B837'><label id='0DD551B837'><select id='0DD551B837'><dt id='0DD551B837'><span id='0DD551B837'></span></dt></select></label></b><u id='0DD551B837'></u>
          <i id='0DD551B837'><strike id='0DD551B837'><tt id='0DD551B837'><pre id='0DD551B837'></pre></tt></strike></i>

          knowledge

          knowledge

          author:focus    Page View:541
          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

          ChatGPT in medicine: STAT answers readers' burning questions
          ChatGPT in medicine: STAT answers readers' burning questions

          MikeReddyforSTATArtificialintelligenceisoftendescribedasablackbox:anunknowable,mysteriousforcethatop

          read more
          Readout Newsletter: Alnylam, Celldex, Roche, Zealand Pharma
          Readout Newsletter: Alnylam, Celldex, Roche, Zealand Pharma

          AdobeWanttostayontopofthescienceandpoliticsdrivingbiotechtoday? Signup togetourbiotechnewsletterinyo

          read more
          BioMarin wins approval for gene therapy to treat hemophilia A
          BioMarin wins approval for gene therapy to treat hemophilia A

          AdobeTheFoodandDrugAdministrationonThursdayapprovedagenetherapytotreatpeoplewithhemophiliaA,aninheri

          read more

          Time to name cancers by genetics, not organ of origin, expert says

          Treatmentsforlungcancercanvarydependingonspecificmutation.Anoncologistarguesthecancersshouldhavediff