One Reporter, Four Families, Six Scientists and the Search to Cure a Rare Disease

A WSJ reporter followed a group of families and scientists trying to accelerate the development of a drug to treat Niemann-Pick Type C disease, a rare and fatal cholesterol metabolism disorder known as NPC that strikes primarily children.

Source: WSJ.com: Health Blog - February 28, 2014 Category: Consumer Health News Authors: Amy Dockser Marcus Tags: Drugs Research Rare diseases Trials Source Type: blogs