Everything seemed fine when the twins were born. Addison and Cassidy Hempel's earliest years were normal — their first words and first steps happened within the normal window for infants. But then both of them experienced troubling symptoms, precipitating multiple medical tests, and then finally, a devastating dual diagnosis: a rare, debilitating and fatal neurodegenerative disease known as Niemann-Pick disease type C.
The Hempel family hoped to put their faith in the scientists who were studying Niemann-Pick disease type C. Yet as they discovered, scientific research moves at a Byzantine pace, and insidious illnesses such as theirs often progress faster than the development of treatments. But as Pulitzer-winning Wall Street Journal writer Amy Dockser Marcus writes in her new book, "We the Scientists: How a Daring Team of Parents and Doctors Forged a New Path for Medicine," that's where the determined work of a motivated population of self-identified "citizen scientists" comes in.
