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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 kutschkeRoger hajjarElizabeth kutschkeBrigham gene cell therapy instituteBaby ben kutschkeMass general brigham geneCell 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 statesDeena beasleyRoger hajjarElizabeth kutschkeRoche evrysdiSitra tauscher wisniewskiAmanda cookEric coxSree chaguturuSara ledwithVasant narasimhanMaha radhakrishnanSteven pearsonStacie dusetzinaBen kutschkeCaroline humer

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 statesRoger hajjarElizabeth kutschkeJerry mendellAmanda cookRussell butterfieldStacie dusetzinaBen kutschkeMaha radhakrishnanVasant narasimhanSitra tauscher wisniewskiRoche evrysdiCaroline humerSteven pearsonEric coxSara ledwith

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

Health: What happens when a $2 million gene therapy is not enough
gdnonline.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from gdnonline.com Daily Mail and Mail on Sunday newspapers.

United statesRoche evrysdiElizabeth kutschkeSree chaguturuVasant narasimhanStacie dusetzinaAmanda cookRoger hajjarRussell butterfieldJerry mendellSitra tauscher wisniewskiBen kutschkeMaha radhakrishnanSteven pearsonInstitute for clinicalInstitute for human data science

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

Insight: What happens when a $2 million gene therapy is not enough
reuters.com - get the latest breaking news, showbiz & celebrity photos, sport news & rumours, viral videos and top stories from reuters.com Daily Mail and Mail on Sunday newspapers.

United statesBen kutschkeRoger hajjarEric coxCaroline humerSteven pearsonRoche evrysdiJerry mendellRussell butterfieldSitra tauscher wisniewskiMaha radhakrishnanElizabeth kutschkeAmanda cookVasant narasimhanStacie dusetzinaSara ledwith

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