Can a wealthy family change the course of a deadly brain disease?

tylermcgraw

This is the model for rare diseases that wouldn’t be profitable for pharmaceutical companies. Spinal muscular atrophy (sma) is another example that comes to mind.

georgeburdell

I know another family like this. One partner still works, but the other one is essentially a full time advocate for an inherited disease that fewer than 100 people in the world are affected by. I don't think much money is involved, but they've changed the narrative about the disease and some researchers are taking them seriously.