How Parents Get Zolgensma, the World’s Most Expensive Drug

Gene therapy is bringing out the best in America’s health-care system—and its worst. Zolgensma, the first systemic gene therapy of its kind that the U.S. Food and Drug Administration has approved, appears to cure in one shot a rare muscle-destroying disease that can be a death sentence for infants and toddlers. (The kids still carry the gene mutation; they just don’t exhibit the eventually fatal symptoms.) It’s also the world’s most expensive medication. Novartis AG set the price at $2.1 million after the FDA approval came down on May 24, and some families have been left scrambling for ways ...