<code id='A9B223E1D7'></code><style id='A9B223E1D7'></style>
    • <acronym id='A9B223E1D7'></acronym>
      <center id='A9B223E1D7'><center id='A9B223E1D7'><tfoot id='A9B223E1D7'></tfoot></center><abbr id='A9B223E1D7'><dir id='A9B223E1D7'><tfoot id='A9B223E1D7'></tfoot><noframes id='A9B223E1D7'>

    • <optgroup id='A9B223E1D7'><strike id='A9B223E1D7'><sup id='A9B223E1D7'></sup></strike><code id='A9B223E1D7'></code></optgroup>
        1. <b id='A9B223E1D7'><label id='A9B223E1D7'><select id='A9B223E1D7'><dt id='A9B223E1D7'><span id='A9B223E1D7'></span></dt></select></label></b><u id='A9B223E1D7'></u>
          <i id='A9B223E1D7'><strike id='A9B223E1D7'><tt id='A9B223E1D7'><pre id='A9B223E1D7'></pre></tt></strike></i>

          Wikipedia

          Wikipedia

          author:explore    Page View:843
          The FDA building -- First Opinion coverage from STAT
          Adobe

          U.S. regulators on Wednesday approved the first treatment for an ultra-rare disease that causes people to grow bone where it otherwise shouldn’t be, extending an option to patients who have been advocating for access to the medicine.

          The treatment, an oral medication that will be marketed as Sohonos, faced some questions about the quality of its data, and was rejected earlier this year by European regulators. But in approving the drug for the treatment of fibrodysplasia ossificans progressiva, or FOP, the Food and Drug Administration concluded that Sohonos’ benefits outweigh its risks.

          advertisement

          “I just closed my eyes and looked up to the skies, like ‘Thank you, God,’” said Suzanne Hollywood, whose teenage son, Joey, has been receiving the drug in a clinical trial. “It’s far from perfect, but I’m thrilled for my son, for his future, and for the kids getting diagnosed who might not have to experience what others had to.”

          Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

          GET STARTED Log In

          explore

          The cancer drug shortage isn’t new — and neither are the solutions
          The cancer drug shortage isn’t new — and neither are the solutions

          PreparingachemotherapytreatmentatDukeCancerCenterinDurham,N.C.GerryBroome/APAyounggirl,maybe5or6year

          read more
          BIO: Crackdown on China pharma industry would threaten U.S. drug supply
          BIO: Crackdown on China pharma industry would threaten U.S. drug supply

          AdobeU.S.legislationthatwouldsevertieswithChinesedrugmakerswouldjeopardizethedrugsupplyformillionsof

          read more
          As demand for Covid shots wanes, Moderna seeks its next success
          As demand for Covid shots wanes, Moderna seeks its next success

          RubyWallauforSTATModernachairmanNoubarAfeyanexudedstarpowerearlierthismonthashecommandedthespotlight

          read more

          FTC falls short on scrutinizing hospital mergers, study says

          AdobeOverthepasttwodecades,hundredsofhospitalmergershaveescapedfederalantitrustscrutinyandledtobothh