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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.

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

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.

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.

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