Category: Rare diseases

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.

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.

Robots to Cure Rare Diseases: A Reddit Ask Me Anything (AMA)

For years, Christopher P. Austin has been trying to develop a way for science to build a better partnership with patients. Dr. Austin and the Journal’s Amy Dockser Marcus are answering questions now.