<code id='D2EA70B7DD'></code><style id='D2EA70B7DD'></style>
    • <acronym id='D2EA70B7DD'></acronym>
      <center id='D2EA70B7DD'><center id='D2EA70B7DD'><tfoot id='D2EA70B7DD'></tfoot></center><abbr id='D2EA70B7DD'><dir id='D2EA70B7DD'><tfoot id='D2EA70B7DD'></tfoot><noframes id='D2EA70B7DD'>

    • <optgroup id='D2EA70B7DD'><strike id='D2EA70B7DD'><sup id='D2EA70B7DD'></sup></strike><code id='D2EA70B7DD'></code></optgroup>
        1. <b id='D2EA70B7DD'><label id='D2EA70B7DD'><select id='D2EA70B7DD'><dt id='D2EA70B7DD'><span id='D2EA70B7DD'></span></dt></select></label></b><u id='D2EA70B7DD'></u>
          <i id='D2EA70B7DD'><strike id='D2EA70B7DD'><tt id='D2EA70B7DD'><pre id='D2EA70B7DD'></pre></tt></strike></i>

          hotspot

          hotspot

          author:leisure time    Page View:69969
          Sarepta - Cambridge -AP
          Kristoffer Tripplaar/Sipa via AP

          To hear Sarepta CEO Doug Ingram tell it, the results Monday afternoon from a large trial of its gene therapy for Duchenne muscular dystrophy marked a clear medical breakthrough. “A massive win,” he told investors, that could lead to its approval for all patients with the fatal muscle-wasting disease regardless of age.

          And yet the rest of the Duchenne community was more cautious in its enthusiasm. The study — the most rigorous test to date of a technology 30 years in the making — had technically failed, as so many had before it.

          advertisement

          “It’s extremely frustrating,” said John Brandsema, a pediatric neurologist at Children’s Hospital of Philadelphia who worked on the study. “When you spend decades trying to bring therapies into the clinic for something so severe and just keep missing over and over again.”

          Get unlimited access to award-winning journalism and exclusive events.

          Subscribe Log In

          knowledge

          Supreme Court strikes down use of affirmative action
          Supreme Court strikes down use of affirmative action

          ActivistsdemonstratedastheSupremeCourtheardoralargumentsonapairofaffirmativeactioncasesinOctober2022

          read more
          Medicare threatens to cut off funding for HCA's Mission hospital
          Medicare threatens to cut off funding for HCA's Mission hospital

          MikeBellemeforSTATFiveyearsafterinvestor-ownedHCAHealthcaretookoveranesteemednonprofithospitalinNort

          read more
          Psychedelics group wrestles with new pharma identity
          Psychedelics group wrestles with new pharma identity

          OliviaGoldhill/STATDENVER—Hecouldhavebeenarockstar,areligiousicon,thewayecstaticapplausefromthousand

          read more

          Medicare threatens to cut off funding for HCA's Mission hospital

          MikeBellemeforSTATFiveyearsafterinvestor-ownedHCAHealthcaretookoveranesteemednonprofithospitalinNort