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A gene therapy treatment for rare SMA syndrome costs $2 million dollars. What happens if it doesn't cure the disease?

Spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. ....

Ben Kutschke , Roger Hajjar , Elizabeth Kutschke , Brigham Gene Cell Therapy Institute , Baby Ben Kutschke , Mass General Brigham Gene , Cell Therapy ,

What happens when a $2 million gene therapy is not enough

(This Aug. 12 story has been corrected to state that ICER's final analysis was that Zolgensma was cost effective at a price of $1.1 million to $1.9 ....

United States , Deena Beasley , Roger Hajjar , Elizabeth Kutschke , Roche Evrysdi , Sitra Tauscher Wisniewski , Amanda Cook , Eric Cox , Sree Chaguturu , Sara Ledwith , Vasant Narasimhan , Maha Radhakrishnan , Steven Pearson , Stacie Dusetzina , Ben Kutschke , Caroline Humer , Russell Butterfield , Jerry Mendell , Brigham Gene Cell Therapy Institute , University Of Utah Health , Institute For Human Data Science , Institute For Clinical , Unitedhealth Group , Nashville Vanderbilt University School Of Medicine , Drug Administration , Baby Ben Kutschke ,

What Happens When a $2 Million Gene Therapy Is Not Enough

Baby Ben Kutschke was diagnosed at 3 months with spinal muscular atrophy, a rare inherited disorder which is the leading genetic cause of death in infancy globally. ....

United States , Roger Hajjar , Elizabeth Kutschke , Jerry Mendell , Amanda Cook , Russell Butterfield , Stacie Dusetzina , Ben Kutschke , Maha Radhakrishnan , Vasant Narasimhan , Sitra Tauscher Wisniewski , Roche Evrysdi , Caroline Humer , Steven Pearson , Eric Cox , Sara Ledwith , Sree Chaguturu , University Of Utah Health , Institute For Clinical , Institute For Human Data Science , Unitedhealth Group , Drug Administration , Brigham Gene Cell Therapy Institute , Nashville Vanderbilt University School Of Medicine , Baby Ben Kutschke , Human Data ,

Health: What happens when a $2 million gene therapy is not enough

Health: What happens when a $2 million gene therapy is not enough
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United States , Roche Evrysdi , Elizabeth Kutschke , Sree Chaguturu , Vasant Narasimhan , Stacie Dusetzina , Amanda Cook , Roger Hajjar , Russell Butterfield , Jerry Mendell , Sitra Tauscher Wisniewski , Ben Kutschke , Maha Radhakrishnan , Steven Pearson , Institute For Clinical , Institute For Human Data Science , University Of Utah Health , Nashville Vanderbilt University School Of Medicine , Brigham Gene Cell Therapy Institute , Unitedhealth Group , Drug Administration , Baby Ben Kutschke , Human Data , Novartis Gene , Mass General Brigham Gene , Cell Therapy ,

Insight: What happens when a $2 million gene therapy is not enough

Insight: What happens when a $2 million gene therapy is not enough
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United States , Ben Kutschke , Roger Hajjar , Eric Cox , Caroline Humer , Steven Pearson , Roche Evrysdi , Jerry Mendell , Russell Butterfield , Sitra Tauscher Wisniewski , Maha Radhakrishnan , Elizabeth Kutschke , Amanda Cook , Vasant Narasimhan , Stacie Dusetzina , Sara Ledwith , Sree Chaguturu , Thomson Reuters Trust Principles , University Of Utah Health , Institute For Clinical , Brigham Gene Cell Therapy Institute , Nashville Vanderbilt University School Of Medicine , Unitedhealth Group , Institute For Human Data Science , Drug Administration , Human Data ,