<code id='BF77079DD3'></code><style id='BF77079DD3'></style>
      • <acronym id='BF77079DD3'></acronym>
      <center id='BF77079DD3'><center id='BF77079DD3'><tfoot id='BF77079DD3'></tfoot></center><abbr id='BF77079DD3'><dir id='BF77079DD3'><tfoot id='BF77079DD3'></tfoot><noframes id='BF77079DD3'>
      • <optgroup id='BF77079DD3'><strike id='BF77079DD3'><sup id='BF77079DD3'></sup></strike><code id='BF77079DD3'></code></optgroup>
        5. <b id='BF77079DD3'><label id='BF77079DD3'><select id='BF77079DD3'><dt id='BF77079DD3'><span id='BF77079DD3'></span></dt></select></label></b><u id='BF77079DD3'></u>
          <i id='BF77079DD3'><strike id='BF77079DD3'><tt id='BF77079DD3'><pre id='BF77079DD3'></pre></tt></strike></i>
           


   

   

   
   
   
   
   
   
   
   
   
   
   
   
   

   
   
   
   
   
   
   
  
           
  
           
  
           
   
           
    
           
     
           
      
           
       
      
           
      
           
       
           
        
           
         
           
           
          
           
         
           
         
           
           
          
           
         
           
          
        
           
       
           
       
           
        
           
        
           
        
           
        
           
       
           
      
           
       
      
           
      
           
       
           
       
           
       
           
       
           
        
           
        
          
          00:00 
        
           
       
           
       
           
      
           
      
           
        
        
        
       
           
        00:00 
         
        00:00 
        LIVE 
       
           
        
       
           
        
           
        
           
        
           
        
           
       
           
       
           
       
           
        
        
        
        
        
        
        
        
        
      
           
       
    
    
      
           
       
           
      
           
     
           
     
           
       
     
           
     
           
      
           
      
           
      
           
      
           
       
           
      
           
      
           
      
           
       
           
       
          
         buffering 
       
           
      
           
      
           
       
           
        
           
        
          
          Replay 
        
           
       
           
      
           
       
      
           
      
           
       
           
       
           
        
           
        
           
        
           
       
           
      
           
      
           
      
           
      
           
      
           
       
           
      
           
     
           
     
           
      
           
     
           
     
           
      
           
       
           
        
           
       
           
       
           
        
          
          LIVE 
        
           
        00:00 
         /  
        00:00 
       
           
       
           
        
           
        
           
        
           
         
           
         
           
         
           
         
           
         
           
         
           
        
           
        
           
        
           
       
           
       
           
        
           
        
          
          LIVE 
        
           
       
           
       
           
        
           
        
          
          CC 
        
           
       
           
       
           
        
           
       
           
       
           
       
           
        
           
        
           
         
           
        
           
       
           
       
           
        
           
       
           
      
           
      
           
       
           
        
           
        
           
         
           
         
           
         
           
          
           
          
           
          
           
         
           
        
           
       
           
      
           
      
           
       
           
     
       
           
    
       
           
       
    
  
       
           
   
       
           
       
       
           
      
           
      
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       
           
       Opacity : 
        
      
           
     
           
     
           
     
           
      
           
  
     
           
    
           
    
           
    
           
     
          
       Share: 
     
           
      
     
           
      
      Copy 
     
           
      
    
           
    
          
      Close 
      
    
           
    
           
      
    
           
    
           
   
           
  
           
  
           
    
    
    
   
           
   
           
 
    
   
           
   
           
   
           
    
           
     
           
      
           
       
           
        
           
          
          
         
          
          entertainment 

          
          
            
          
          

           
           
        
           
       
           
      
           
     
           
    
           
    
           
     
           
      
           
      
           

                       
          entertainment  
          
                                   
                 
                                      author：explore   
                                        
                                      
                                      -
 
browse：12
                                  
                                    
          
                                    
            
          
 
          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															
           
 
 
          

                              
          
                                 Previous article：

                                
                                          Heart attack and stroke risk calculators need revising
                                
                            
          Next article：  
                                      
                                          In science, is the line between industry and academia shifting?
                                                                    
          

      
           
      
           
        
           
                                               
        
           entertainment 
           
                   
      
           
  
  
          
    
          
        
          
            
          
                                                     
                
          explore
          
                            
          
        
          
        
    
          

          

    

      
           
     
           


   
    
           
   
           
  
 

  
           
    
           
           
           
            
            Back To Top 
           
           
           
           

            
           
                                                                    
                 
          entertainment
           
                                      
            
           
          
           
                                                          
             
          hotspot
           
                             
            
           
            
           

                                                         
             
          hotspot
           
               
              
            
           
            
           
                                                         
             
          leisure time
           
                             
      
           
 
     
           
     
           
      
          
              PackagesofmifepristonetabletsaredisplayedatafamilyplanningclinicinRockville,Md.AnnaMoneymaker/GettyI
             
           
      
          
        © 2013-2023.Company name All rights reserved.Site Map 
         WSS-More Templates  
             Guangdong ICP724name