<code id='960C6EDD3A'></code><style id='960C6EDD3A'></style>
    • <acronym id='960C6EDD3A'></acronym>
      <center id='960C6EDD3A'><center id='960C6EDD3A'><tfoot id='960C6EDD3A'></tfoot></center><abbr id='960C6EDD3A'><dir id='960C6EDD3A'><tfoot id='960C6EDD3A'></tfoot><noframes id='960C6EDD3A'>

    • <optgroup id='960C6EDD3A'><strike id='960C6EDD3A'><sup id='960C6EDD3A'></sup></strike><code id='960C6EDD3A'></code></optgroup>
        1. <b id='960C6EDD3A'><label id='960C6EDD3A'><select id='960C6EDD3A'><dt id='960C6EDD3A'><span id='960C6EDD3A'></span></dt></select></label></b><u id='960C6EDD3A'></u>
          <i id='960C6EDD3A'><strike id='960C6EDD3A'><tt id='960C6EDD3A'><pre id='960C6EDD3A'></pre></tt></strike></i>

          comprehensive

          comprehensive

          author:comprehensive    Page View:1942
          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

          Activists slam private health insurers over coverage restrictions
          Activists slam private health insurers over coverage restrictions

          ProtestorsoutsideAmerica'sHealthInsurancePlansheadquarters.CourtesyPeople'sActionWASHINGTON—Hun

          read more
          Boston competes with Raleigh
          Boston competes with Raleigh

          PatGreenhouse/GlobeStaffMORRISVILLE,N.C.—TenminutesfromtheRaleighairport,thefutureofbiotechisunderco

          read more
          In age of alternative facts, a scholarly course on calling out crap
          In age of alternative facts, a scholarly course on calling out crap

          Screenshotviacallingbullshit.orgTiredofalternativefacts,fakenews,andbreathlesshyperbole,twoprofessor

          read more

          Streamlined flu vaccines recommended by FDA advisory panel

          AfluvaccinesyringerestsonatableatavaccinationclinicinLakewood,Calif.MarioTama/GettyImagesWhenAmerica