Saw this one in the WSJ:
https://themighty.com/author/luke-rosen/
https://www.wsj.com/articles/on-the-way-to-a-rare-disease-cu...
And this one on ABC News tonight:
https://donate.curecmt4j.org/campaign/cure-cmt4j-advancing-g...
https://www.fda.gov/ForIndustry/DevelopingProductsforRareDis...
The problem is this results in drugs that cost a fortune and still only helps a small percentage of sufferers, like the $300k drug for CF that only helps 5% of CF patients.
http://www.cbc.ca/beta/news/canada/nova-scotia/cystic-fibros...
Saw this one in the WSJ:
https://themighty.com/author/luke-rosen/
https://www.wsj.com/articles/on-the-way-to-a-rare-disease-cu...
And this one on ABC News tonight:
https://donate.curecmt4j.org/campaign/cure-cmt4j-advancing-g...