<code id='2737B6131E'></code><style id='2737B6131E'></style>
      • <acronym id='2737B6131E'></acronym>
      <center id='2737B6131E'><center id='2737B6131E'><tfoot id='2737B6131E'></tfoot></center><abbr id='2737B6131E'><dir id='2737B6131E'><tfoot id='2737B6131E'></tfoot><noframes id='2737B6131E'>
      • <optgroup id='2737B6131E'><strike id='2737B6131E'><sup id='2737B6131E'></sup></strike><code id='2737B6131E'></code></optgroup>
        5. <b id='2737B6131E'><label id='2737B6131E'><select id='2737B6131E'><dt id='2737B6131E'><span id='2737B6131E'></span></dt></select></label></b><u id='2737B6131E'></u>
          <i id='2737B6131E'><strike id='2737B6131E'><tt id='2737B6131E'><pre id='2737B6131E'></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 

          
          
            
          
          

           
           
        
           
       
           
      
           
     
           
    
           
    
           
     
           
      
           
      
           

                       
          comprehensive  
          
                                   
                 
                                      author：knowledge   
                                        
                                      
                                      -
 
browse：4
                                  
                                    
          
                                    
            
          
 
          Sarepta - Cambridge -AP
          Kristoffer Tripplaar/Sipa via AP
          The Food and Drug Administration granted conditional approval Thursday to the first gene therapy for Duchenne muscular dystrophy. Regulators restricted the treatment to younger patients, with additional data required to broaden its use.
          The gene therapy, called Elevydis, is made by Sarepta Therapeutics. The company will charge $3.2 million for the treatment, making it the U.S.’s second most expensive drug, behind a recently approved gene therapy for hemophilia. CEO Doug Ingram said on a conference call the price was below what a recently published company-funded analysis found would be cost-effective.
          advertisement
          “The approval of Elevidys is a watershed moment for the treatment of Duchenne. Elevydis is the first and only gene therapy approved for Duchenne, and this approval brings us closer to our goal of bringing forward a treatment that provides the potential to alter the trajectory of this degenerative disease,” Ingram said.
          Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!					
          GET STARTED													Log In											
           
 
 
          

                              
          
                                 Previous article：

                                
                                          Drug repurposing or repositioning? The language matters
                                
                            
          Next article：  
                                      
                                          Readout Newsletter: Alnylam, Celldex, Roche, Zealand Pharma
                                                                    
          

      
           
      
           
        
           
                                               
        
           hotspot 
           
                   
      
           
  
  
          
    
          
        
          
            
          
                                                     
                
          leisure time
          
                            
          
        
          
        
    
          

          

    

      
           
     
           


   
    
           
   
           
  
 

  
           
    
           
           
           
            
            Back To Top 
           
           
           
           

            
           
                                                                    
                 
          explore
           
                                      
            
           
          
           
                                                          
             
          focus
           
                             
            
           
            
           

                                                         
             
          hotspot
           
               
              
            
           
            
           
                                                         
             
          explore
           
                             
      
           
 
     
           
     
           
      
          
              Alabama'slethalinjectionchamberatHolmanCorrectionalFacilityinAtmore,Ala.DaveMartin/APAftertherecentn
             
           
      
          
        © 2013-2023.Company name All rights reserved.Site Map 
         WSS-More Templates  
             Guangdong ICP9name