<code id='E4312A79FB'></code><style id='E4312A79FB'></style>
    • <acronym id='E4312A79FB'></acronym>
      <center id='E4312A79FB'><center id='E4312A79FB'><tfoot id='E4312A79FB'></tfoot></center><abbr id='E4312A79FB'><dir id='E4312A79FB'><tfoot id='E4312A79FB'></tfoot><noframes id='E4312A79FB'>

    • <optgroup id='E4312A79FB'><strike id='E4312A79FB'><sup id='E4312A79FB'></sup></strike><code id='E4312A79FB'></code></optgroup>
        1. <b id='E4312A79FB'><label id='E4312A79FB'><select id='E4312A79FB'><dt id='E4312A79FB'><span id='E4312A79FB'></span></dt></select></label></b><u id='E4312A79FB'></u>
          <i id='E4312A79FB'><strike id='E4312A79FB'><tt id='E4312A79FB'><pre id='E4312A79FB'></pre></tt></strike></i>

          focus

          focus

          author:Wikipedia    Page View:3398
          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

          Anesthesiologist group: stop taking Ozempic before surgery
          Anesthesiologist group: stop taking Ozempic before surgery

          EspeciallyinthefirstweeksoftakingdrugslikeOzempic,foodstayslongerinthestomach—aprobleminsurgeries.Ad

          read more
          Bernie brings in new pharma CEOs to talk drug prices
          Bernie brings in new pharma CEOs to talk drug prices

          Sen.BernieSanders(I-Vt.)Moneymaker/GettyImagesYou’rereadingthewebeditionofD.C.Diagnosis,STAT’stwice-

          read more
          Wildfire smoke exposes gaps in outdoor worker protections
          Wildfire smoke exposes gaps in outdoor worker protections

          ApersonwaitingforthesubwaywearsafilteredmaskassmokyhazefromwildfiresinCanadablanketsaneighborhoodinN

          read more

          Readout Newsletter: Acadia, Eli Lilly, Madrigal Pharma updates

          DarronCummings/APWanttostayontopofthescienceandpoliticsdrivingbiotechtoday? Signup togetourbiotechne