A terrible disease, children at risk, and a promising treatment that’s about to vanish: Part II
Editor’s note: This is Part II of a two-part story. You can read Part I, which was originally published on April 14, by clicking here.
A rare childhood disorder, Niemann-Pick Disease type C (NPC), affects just a few dozen children in the United States each year. One of a number of lysosomal storage disorders, the disease affects the ability to metabolize cholesterol, and results in widespread damage over time.