<code id='5F3A5B3F34'></code><style id='5F3A5B3F34'></style>
    • <acronym id='5F3A5B3F34'></acronym>
      <center id='5F3A5B3F34'><center id='5F3A5B3F34'><tfoot id='5F3A5B3F34'></tfoot></center><abbr id='5F3A5B3F34'><dir id='5F3A5B3F34'><tfoot id='5F3A5B3F34'></tfoot><noframes id='5F3A5B3F34'>

    • <optgroup id='5F3A5B3F34'><strike id='5F3A5B3F34'><sup id='5F3A5B3F34'></sup></strike><code id='5F3A5B3F34'></code></optgroup>
        1. <b id='5F3A5B3F34'><label id='5F3A5B3F34'><select id='5F3A5B3F34'><dt id='5F3A5B3F34'><span id='5F3A5B3F34'></span></dt></select></label></b><u id='5F3A5B3F34'></u>
          <i id='5F3A5B3F34'><strike id='5F3A5B3F34'><tt id='5F3A5B3F34'><pre id='5F3A5B3F34'></pre></tt></strike></i>

          explore

          explore

          author:comprehensive    Page View:2
          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

          Dobbs anniversary: the lost opportunity of abortion as health care
          Dobbs anniversary: the lost opportunity of abortion as health care

          NathanHoward/GettyImagesReflectingonthisfirstanniversaryoftheSupremeCourt’sdecisioninDobbstooverturn

          read more
          RSV injection headlines need to be accurate for good uptake
          RSV injection headlines need to be accurate for good uptake

          STAT/AstraZenecaviaAPAstheoristsfromSamuelColeridgetoKennethBurkehavenoted,languageoftendoesourthink

          read more
          Apple is now the first public company to be valued at $3 trillion
          Apple is now the first public company to be valued at $3 trillion

          6:09FILE-AnApplelogoadornsthefacadeofthedowntownBrooklynApplestoreonMarch14,2020,inNewYork.Applebeca

          read more

          Nobel honors Karikó and Weissman's enduring partnership

          DrewWeissman(left)andKatalinKarikó,whoselongtimepartnershipwasrecognizedwithaNobelPrizeonMonday.Pegg