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.
Category: Research
Rare Diseases and the Search for a Cure: A Reddit AMA
Dr. Christopher Austin and the Journal’s Health reporter Amy Dockser Marcus took questions about treatments for rare disease during a Reddit AMA.
