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          knowledge 

          
          
            
          
          

           
           
        
           
       
           
      
           
     
           
    
           
    
           
     
           
      
           
      
           

                       
          Wikipedia  
          
                                   
                 
                                      author：explore   
                                        
                                      
                                      -
 
browse：74
                                  
                                    
          
                                    
            
          
 
          Colorful broken DNA helix genome. -- health coverage from STAT
          Adobe 
          In 2020, as biotech stocks surged amid the pandemic, a startup called Taysha Gene Therapies raised over $300 million off an audacious promise: It was going to license and develop gene therapies for at least 18 different rare and serious neurological diseases.
          The effort brought hope for many rare disease families, some of whom had raised money to fund the UT-Southwestern scientist who designed the treatments after they saw no other options for their children.
          advertisement
          Last week, the grand plan took perhaps a final blow. Taysha announced it would discontinue development of a treatment for giant axonal neuropathy, or GAN, a fatal, degenerative disease known to affect fewer than 100 people worldwide. The FDA, Taysha said, had requested an additional clinical trial that would be infeasible.
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