Description

A Pulitzer Prizewinning reporters moving narrative of a group of patient advocates who are revolutionizing the way medical research is conducted

For more than half a century medical advances have been driven by investigators launching experiments inside labs Science is often conducted in isolation and geared toward the long view This is the story of a group of people who tried to force the lab doors open parents whose children had been diagnosed with a rare and fatal genetic condition known as NiemannPick disease type C The disease prevents cells from processing cholesterol which leads to the progressive loss of the brains and the bodys ability to function Recognizing that there would never be a treatment in time to save their children if things stayed the same the parents set up a collaboration with researchers and doctors in search of a cure

Reconciling different views of science took work The parents doctors and researchers didnt always agreeamong themselves or with each other But together they endeavored toaccelerate the development of new drugs The parents became citizen scientists identifying promising new treatments and helping devise experiments They recorded dataaboutthe children and coauthored scientific papers sharing findings They engaged directly with the FDA at each step of the drug approval process Along the way they advanced the radical idea that science must belong to us all

Amy Dockser Marcus shows what happens when a community joins forces with doctors and researchers to try to save childrens lives Their extraordinary social experiment reveals new pathways for treating disease and conducting research Science may be forever changed