<code id='E643FADE1B'></code><style id='E643FADE1B'></style>
    • <acronym id='E643FADE1B'></acronym>
      <center id='E643FADE1B'><center id='E643FADE1B'><tfoot id='E643FADE1B'></tfoot></center><abbr id='E643FADE1B'><dir id='E643FADE1B'><tfoot id='E643FADE1B'></tfoot><noframes id='E643FADE1B'>

    • <optgroup id='E643FADE1B'><strike id='E643FADE1B'><sup id='E643FADE1B'></sup></strike><code id='E643FADE1B'></code></optgroup>
        1. <b id='E643FADE1B'><label id='E643FADE1B'><select id='E643FADE1B'><dt id='E643FADE1B'><span id='E643FADE1B'></span></dt></select></label></b><u id='E643FADE1B'></u>
          <i id='E643FADE1B'><strike id='E643FADE1B'><tt id='E643FADE1B'><pre id='E643FADE1B'></pre></tt></strike></i>

          fashion

          fashion

          author:knowledge    Page View:93
          A Exterior of the new Ultragenyx biomanufacturing plant. -- biotech coverage from STAT
          Jonathan Wiggs /Globe Staff

          Ultragenyx said Thursday afternoon that its gene therapy for a rare liver ailment, glycogen disease type 1A, succeeded in a Phase 3 trial, setting up a potential approval.

          Patients with GSD1a, as the condition is often known, have a genetic mutation that prevents them from adequately maintaining blood sugar levels. Once considered fatal, GSD1a can now be controlled with regular doses of cornstarch. But if patients miss a dose, there can be significant, even life-threatening, complications.

          advertisement

          In the 49-person, randomized study, patients who received the Ultragenyx drug were able to take 41% less cornstarch after 48 weeks than they did at the start. Patients on placebo took only 10% less. The difference was statistically significant. Five patients were not included in the analysis, as three dropped out and the company didn’t have 48-week data from two.

          STAT+ Exclusive Story

          Already have an account? Log in

          STAT+

          This article is exclusive to STAT+ subscribers

          Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

          Already have an account? Log in

          Already have an account? Log in

          Monthly

          $39

          Totals $468 per year

          $39/month Get Started

          Totals $468 per year

          Starter

          $30

          for 3 months, then $39/month

          $30 for 3 months Get Started

          Then $39/month

          Annual

          $399

          Save 15%

          $399/year Get Started

          Save 15%

          11+ Users

          Custom

          Savings start at 25%!

          Request A Quote Request A Quote

          Savings start at 25%!

          2-10 Users

          $300

          Annually per user

          $300/year Get Started

          $300 Annually per user

          View All Plans

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

          Subscribe Log In

          focus

          What does generative AI mean for health care? We asked experts
          What does generative AI mean for health care? We asked experts

          MikeReddyforSTATHealthcarecompaniesareracingtoincorporategenerativeAItoolsintotheirproductpipelinesa

          read more
          Pharma patents targeted by Biden in move to slash drug prices
          Pharma patents targeted by Biden in move to slash drug prices

          PresidentBidenMANDELNGAN/AFPviaGettyImagesWASHINGTON—TheWhiteHouseisthrowingitssupportbehindacontrov

          read more
          Duchenne breakthrough therapy leaves behind pioneering families
          Duchenne breakthrough therapy leaves behind pioneering families

          DuchennemusculardystrophyDr.EdwinP.Ewing,Jr./CDCPatFurlongwassittinginherhomeofficeinMiddletown,Ohio

          read more

          Federal weight loss drug coverage is 30 years out of date

          ApharmacistholdsaboxofWegovyatapharmacyinProvo,Utah.BloombergphotobyGeorgeFreyFewmedicationshavecapt