Who deserves to get a potentially life-saving treatment? How severe does the disease have to be? At what age should someone become ineligible?
These questions are tormenting families trying to obtain Zolgensma, the world’s most expensive drug. The $2.1 million gene therapy was approved in May for children up to the age of 2 with spinal muscular atrophy, a rare disease that kills more infants than any other inherited disorder.