<code id='B40DCFF3C7'></code><style id='B40DCFF3C7'></style>
    • <acronym id='B40DCFF3C7'></acronym>
      <center id='B40DCFF3C7'><center id='B40DCFF3C7'><tfoot id='B40DCFF3C7'></tfoot></center><abbr id='B40DCFF3C7'><dir id='B40DCFF3C7'><tfoot id='B40DCFF3C7'></tfoot><noframes id='B40DCFF3C7'>

    • <optgroup id='B40DCFF3C7'><strike id='B40DCFF3C7'><sup id='B40DCFF3C7'></sup></strike><code id='B40DCFF3C7'></code></optgroup>
        1. <b id='B40DCFF3C7'><label id='B40DCFF3C7'><select id='B40DCFF3C7'><dt id='B40DCFF3C7'><span id='B40DCFF3C7'></span></dt></select></label></b><u id='B40DCFF3C7'></u>
          <i id='B40DCFF3C7'><strike id='B40DCFF3C7'><tt id='B40DCFF3C7'><pre id='B40DCFF3C7'></pre></tt></strike></i>

          comprehensive

          comprehensive

          author:leisure time    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

          explore

          Biden outlines 'new path' to provide student loan relief after Supreme Court rejection
          Biden outlines 'new path' to provide student loan relief after Supreme Court rejection

          6:16PresidentJoeBidenspeaksintheRooseveltRoomoftheWhiteHouse,June30,2023.EvanVucci/APPresidentJoeBid

          read more
          Eli Lilly expects to double planned employment in Boston center
          Eli Lilly expects to double planned employment in Boston center

          Anartist'srenderingofthebuilding—currentlyunderconstructionalongBoston'sFortPointChannel—whereEliLil

          read more
          Duchenne breakthrough therapy leaves behind pioneering families
          Duchenne breakthrough therapy leaves behind pioneering families

          DuchennemusculardystrophyDr.EdwinP.Ewing,Jr./CDCPatFurlongwassittinginherhomeofficeinMiddletown,Ohio

          read more

          Studies point to risks of excluding people with obesity from drug trials

          AdobePeoplewithobesityoftengounderrepresentedindrugdevelopmenttrials,acriticalgapthatresearcherssayl