<code id='24AB222F1A'></code><style id='24AB222F1A'></style>
    • <acronym id='24AB222F1A'></acronym>
      <center id='24AB222F1A'><center id='24AB222F1A'><tfoot id='24AB222F1A'></tfoot></center><abbr id='24AB222F1A'><dir id='24AB222F1A'><tfoot id='24AB222F1A'></tfoot><noframes id='24AB222F1A'>

    • <optgroup id='24AB222F1A'><strike id='24AB222F1A'><sup id='24AB222F1A'></sup></strike><code id='24AB222F1A'></code></optgroup>
        1. <b id='24AB222F1A'><label id='24AB222F1A'><select id='24AB222F1A'><dt id='24AB222F1A'><span id='24AB222F1A'></span></dt></select></label></b><u id='24AB222F1A'></u>
          <i id='24AB222F1A'><strike id='24AB222F1A'><tt id='24AB222F1A'><pre id='24AB222F1A'></pre></tt></strike></i>

          hotspot

          hotspot

          author:leisure time    Page View:68222
          Gene therapy helped Conner Curran, right, walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. -- Biotech coverage from STAT
          Gene therapy helped Conner Curran (at right) walk, but as the drug fades, his family is supporting scientific efforts to allow him and other boys get a second dose. Courtesy Jess & Chris Curran

          In the beginning, Conner Curran ran up the stairs.

          It was a miraculous moment for him and his parents, Jess and Chris Curran. Just months before, contractors were in their New York home to install a motorized lift, while Jess tearfully explained that it wasn’t for a grandparent but for their 7-year-old son, who suffered from Duchenne muscular dystrophy and could barely haul himself up the steps.

          advertisement

          Then Conner was accepted as the first patient in Pfizer’s Duchenne gene therapy trial. Three weeks later, he could bound to the second floor. Soon, he could walk the two-plus miles to Goldberg’s, a local bagel shop. “I couldn’t do that before,” he told NPR in 2020. 

          Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

          GET STARTED Log In

          focus

          Continuity nursing helped my family in our darkest PICU moments
          Continuity nursing helped my family in our darkest PICU moments

          EssayauthorSarahMcCarthycuddleswithherdaughterMolly.CourtesySarahMcCarthyTheventilatoralarmwokemeat3

          read more
          STAT Virtual Event: How to Increase Diversity in Clinical Trials
          STAT Virtual Event: How to Increase Diversity in Clinical Trials

          Editor’snote:Alivestreamoftheeventwillbeembeddedbelowat1p.m.ET. Howdoweensurethepopulationinastudybe

          read more
          Trump demands the U.S. pay no more for drugs than other countries … again
          Trump demands the U.S. pay no more for drugs than other countries … again

          EthanMiller/GettyImagesWASHINGTON—FormerPresidentTrumpisbacktocampaigningfortyingMedicaredrugpricest

          read more

          ALS groups 'won't be played again' by the FDA, drug sponsors

          MattScozzariholdshisgirlfriendSabrinaParker'shandduringafriendshipceremonyinJacksonville,N.C.,inNove