<code id='5C0C805D3D'></code><style id='5C0C805D3D'></style>
    • <acronym id='5C0C805D3D'></acronym>
      <center id='5C0C805D3D'><center id='5C0C805D3D'><tfoot id='5C0C805D3D'></tfoot></center><abbr id='5C0C805D3D'><dir id='5C0C805D3D'><tfoot id='5C0C805D3D'></tfoot><noframes id='5C0C805D3D'>

    • <optgroup id='5C0C805D3D'><strike id='5C0C805D3D'><sup id='5C0C805D3D'></sup></strike><code id='5C0C805D3D'></code></optgroup>
        1. <b id='5C0C805D3D'><label id='5C0C805D3D'><select id='5C0C805D3D'><dt id='5C0C805D3D'><span id='5C0C805D3D'></span></dt></select></label></b><u id='5C0C805D3D'></u>
          <i id='5C0C805D3D'><strike id='5C0C805D3D'><tt id='5C0C805D3D'><pre id='5C0C805D3D'></pre></tt></strike></i>

          fashion

          fashion

          author:knowledge    Page View:894
          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

          Alkermes shareholders re
          Alkermes shareholders re

          MarkLennihan/APAlkermesshareholdersvotedThursdaytore-electallofthedrugmaker’scurrentdirectors,ending

          read more
          Some dementia cases could be undiagnosed liver disease
          Some dementia cases could be undiagnosed liver disease

          AdobeThedescentintodementiacanfeelliketraversingaminefield,comingacrossnewsymptomswithoutthehopeofac

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

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

          read more

          Time to name cancers by genetics, not organ of origin, expert says

          Treatmentsforlungcancercanvarydependingonspecificmutation.Anoncologistarguesthecancersshouldhavediff