<code id='9E29B2CE68'></code><style id='9E29B2CE68'></style>
    • <acronym id='9E29B2CE68'></acronym>
      <center id='9E29B2CE68'><center id='9E29B2CE68'><tfoot id='9E29B2CE68'></tfoot></center><abbr id='9E29B2CE68'><dir id='9E29B2CE68'><tfoot id='9E29B2CE68'></tfoot><noframes id='9E29B2CE68'>

    • <optgroup id='9E29B2CE68'><strike id='9E29B2CE68'><sup id='9E29B2CE68'></sup></strike><code id='9E29B2CE68'></code></optgroup>
        1. <b id='9E29B2CE68'><label id='9E29B2CE68'><select id='9E29B2CE68'><dt id='9E29B2CE68'><span id='9E29B2CE68'></span></dt></select></label></b><u id='9E29B2CE68'></u>
          <i id='9E29B2CE68'><strike id='9E29B2CE68'><tt id='9E29B2CE68'><pre id='9E29B2CE68'></pre></tt></strike></i>

          knowledge

          knowledge

          author:leisure time    Page View:256
          Scorecard
          Ryan Pierse/Getty Images

          Here is STAT’s biotech scorecard, our regular ledger of stock-moving biotech events, for the fourth quarter:

          The Food and Drug Administration’s decision in June to grant conditional approval to a gene therapy for Duchenne muscular dystrophy was a watershed moment for the treatment of the neuromuscular disease, and a big win for its maker, Sarepta Therapeutics. Confirming the gene therapy’s benefit — and potentially broadening its use to include older boys — will be determined by a Phase 3 clinical trial that will read out later this quarter.

          advertisement

          Pfizeris developing its own gene therapy for Duchenne in a fully enrolled Phase 3 study with final results expected in 2024. But before the verdict is revealed, Pfizer intends to conduct an interim analysis later this quarter. The most likely outcome of this early look will be a determination to continue the study to its completion. But surprises — including an early stop for overwhelming efficacy — can’t be ruled out.

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

          Subscribe Log In

          explore

          Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections
          Indiana Supreme Court upholds abortion ban, says state constitution gives only limited protections

          FILE-Abortion-rightsprotestersfillIndianaStatehousecorridorsandcheeroutsidelegislativechambers,Frida

          read more
          Readout Newsletter: The latest at Gilead, Catalent, and the NIH
          Readout Newsletter: The latest at Gilead, Catalent, and the NIH

          Wanttostayontopofthescienceandpoliticsdrivingbiotechtoday? Signup togetourbiotechnewsletterinyourinb

          read more
          ChatGPT in medicine: STAT answers readers' burning questions
          ChatGPT in medicine: STAT answers readers' burning questions

          MikeReddyforSTATArtificialintelligenceisoftendescribedasablackbox:anunknowable,mysteriousforcethatop

          read more

          Why medical students are struggling with mental health

          Forthemanyhigh-achievingperfectionistswhoentermedicalschooleachyear,thestakesoftheirsuccesshavenever