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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 ,

When a $2 million gene therapy is not enough

Ben is one of a growing number of patients with spinal muscular atrophy whose doctors are turning to additional drugs in addition to gene therapy. ....

Los Angeles , 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 News Roundup: US Supreme Court halts Purdue Pharma bankruptcy settlement pending review; Uzbekistan starts trial over 65 child deaths linked to contaminated cough syrups and more

Health News Roundup: US Supreme Court halts Purdue Pharma bankruptcy settlement pending review; Uzbekistan starts trial over 65 child deaths linked to contaminated cough syrups and more
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