They Created a Drug for Susannah. What About Millions of Other Patients?

Susannah Rosen, 8, spent much of her childhood in hospitals in New York City as doctors documented the gradual loss of her ability to stand, walk and see.

But on a visit this October [to Dr. Jennifer Bain, a pediatric neurologist at NewYork-Presbyterian Morgan Stanley Children’s Hospital in Manhattan], her parents thought for the first time that she might leave the hospital better off than before. That’s when surgeons infused a drug into her spine to fix the ultrarare genetic glitch that had vexed her nervous system since infancy. [read more]

Source: The New York Times