A terrible disease, children at risk, and a promising treatment that’s about to vanish: Part I
The U.S. Food and Drug Administration (FDA) has a specific classification for what is known as a “rare pediatric disease.” Every condition that meets that designation is, by definition, horrible. These are the diseases that most families are blissfully unaware of, while for others the names of these diseases—Severe Combined Immunodeficiency Disease, CANDLE Syndrome, Pompe Disease—become a dark drumbeat that sounds behind each moment of their lives.





























