WASHINGTON, D.C. (NBC) – Federal health officials have approved the most expensive medicine in the world.
The FDA has approved the gene therapy Zolgensma to treat spinal muscular atrophy.
Children with this rare, inherited disease are unable to sit or breathe without assistance and only live a few years.
Clinical trials show Zolgensma improved breathing and motor function in most patients. But the treatment comes with a hefty price tag.
A one-time injection will cost $2.1 million dollars and will be available within two weeks.
Zolgensma is manufactured by Novartis.
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